MedWorm: Gene Therapy

Interference of CD40L-Mediated Tumor Immunotherapy by Oncolytic Vesicular Stomatitis Virus

Human Gene Therapy — Fri, 19 Mar 2010 03:03:54 +0100

Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)

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Critical Drug Information Not Reaching Prescribers; Guidance for REMS; Stem Cell Trial for ALS Scheduled; Orphan Drug Status for a Gene Therapy; Safe Use Initiative from FDA; Antigenics Withdraws European Oncophage Application

Biotechnology Law Report — Fri, 19 Mar 2010 00:21:06 +0100

Biotechnology Law Report Dec 2009, Vol. 28, No. 6: 717-718. (Source: Biotechnology Law Report)

Gene Sequences May Make You Unique

MedlinePlus Health News — Thu, 18 Mar 2010 18:00:00 +0100

Focusing on genes alone will yield incomplete picture, scientists say Source: HealthDay Related MedlinePlus Page: Genes and Gene Therapy (Source: MedlinePlus Health News)

B cell-specific lentiviral gene therapy leads to sustained B-cell functional recovery in a murine model of X-linked agammaglobulinemia

Blood — Thu, 18 Mar 2010 16:02:03 +0100

The immunodeficiency disorder, X-linked agammaglobulinemia (XLA), results from mutations in the gene encoding Bruton tyrosine kinase (Btk). Btk is required for pre-B cell clonal expansion and B-cell antigen receptor signaling. XLA patients lack mature B cells and immunoglobulin and experience recurrent bacterial infections only partially mitigated by life-long antibody replacement therapy. In pursuit of definitive therapy for XLA, we tested ex vivo gene therapy using a lentiviral vector (LV) containing the immunoglobulin enhancer (Eµ) and Igβ (B29) minimal promoter to drive B lineage–specific human Btk expression in Btk/Tec–/– mice, a strain that reproduces the features of human XLA. After transplantation of EµB29-Btk-LV–transduced stem cells, treat...

Future Prospects and Challenges of Antiangiogenic Cancer Gene Therapy

Human Gene Therapy — Thu, 18 Mar 2010 03:40:14 +0100

Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)

Cardiac Gene Therapy: Optimization of Gene Delivery Techniques In Vivo

Human Gene Therapy — Thu, 18 Mar 2010 03:29:06 +0100

Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)

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Prolonged systemic circulation of chimeric oncolytic adenovirus Ad5/3-Cox2L-D24 in patients with metastatic and refractory solid tumors

Gene Therapy — Thu, 18 Mar 2010 00:00:00 +0100

Prolonged systemic circulation of chimeric oncolytic adenovirus Ad5/3-Cox2L-D24 in patients with metastatic and refractory solid tumors Gene Therapy advance online publication, March 18, 2010. doi:10.1038/gt.2010.17 Authors: S Pesonen, P Nokisalmi, S Escutenaire, M Särkioja, M Raki, V Cerullo, L Kangasniemi, L Laasonen, C Ribacka, K Guse, E Haavisto, M Oksanen, M Rajecki, A Helminen, A Ristimäki, A Karioja-Kallio, E Karli, T Kantola, G Bauerschmitz, A Kanerva, T Joensuu & A Hemminki (Source: Gene Therapy)

Feline leukemia virus integrase and capsid packaging functions do not change the insertion profile of standard Moloney retroviral vectors

Gene Therapy — Thu, 18 Mar 2010 00:00:00 +0100

Authors: J-Y Métais, S Topp, R T Doty, B Borate, A-D Nguyen, T G Wolfsberg, J L Abkowitz & C E Dunbar (Source: Gene Therapy)

Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency

Gene Therapy — Thu, 18 Mar 2010 00:00:00 +0100

Authors: J Pang, S E Boye, B Lei, S L Boye, D Everhart, R Ryals, Y Umino, B Rohrer, J Alexander, J Li, X Dai, Q Li, B Chang, R Barlow & W W Hauswirth (Source: Gene Therapy)

Indirect imaging of cardiac-specific transgene expression using a bidirectional two-step transcriptional amplification strategy

Gene Therapy — Thu, 18 Mar 2010 00:00:00 +0100

Authors: I Y Chen, O Gheysens, S Ray, Q Wang, P Padmanabhan, R Paulmurugan, A M Loening, M Rodriguez-Porcel, J K Willmann, A Y Sheikh, C H Nielsen, G Hoyt, C H Contag, R C Robbins, S Biswal, J C Wu & S S Gambhir (Source: Gene Therapy)

Retraction

Human Gene Therapy — Wed, 17 Mar 2010 20:39:54 +0100

Human Gene Therapy Mar 2010, Vol. 21, No. 3: 363-363. (Source: Human Gene Therapy)

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Clinical Impact of Suicide Gene Therapy in Allogeneic Hematopoietic Stem Cell Transplantation

Human Gene Therapy — Wed, 17 Mar 2010 20:39:16 +0100

Human Gene Therapy Mar 2010, Vol. 21, No. 3: 241-250. (Source: Human Gene Therapy)

Tests for Genes Don't Predict Breast Cancer Better

MedlinePlus Health News — Wed, 17 Mar 2010 20:39:09 +0100

Studying genes linked to breast cancer may someday lead to better treatments, but they do little to improve a doctor's ability to predict who is likely to develop a tumor, researchers reported on Wednesday.Source: Reuters Health Related MedlinePlus Pages: Breast Cancer, Genes and Gene Therapy (Source: MedlinePlus Health News)

The Wire

Human Gene Therapy — Wed, 17 Mar 2010 20:36:33 +0100

Human Gene Therapy Mar 2010, Vol. 21, No. 3: 238-240. (Source: Human Gene Therapy)

Overexpression of c-erbB-2 and loss of p16 have molecular diagnostic relevance but no prognostic value in lung cancer

Medical Oncology — Wed, 17 Mar 2010 15:51:54 +0100

This study was designed to evaluate the expression of C-erbB-2 and p16 in lung cancers using tissue microarray technology and to determine their clinical and pathological significance. Immunohistochemical C-erbB-2 and p16 expressions and their associations with clinical and pathological features were analyzed in two tissue microarrays. The membranous and cytoplasmic expression rates of C-erbB-2 were 40.5 and 66.5% in non-small cell lung cancers (NSCLCs), and 0 and 9.5% in small cell lung cancers (SCLCs), respectively. The nuclear and cytoplasmic expression rates of p16 were 11.5 and 32.2% in NSCLs, and 45 and 80% in SCLCs, respectively. The cytoplasmic expression of both C-erbB-2 and p16 was more frequent than the membranous expression of C-erbB-2 and the nuclear expression of p16. T...

Mesothelioma: an inviting but challenging target for gene therapy.

American Journal of Respiratory Cell and Molecular Biology — Wed, 17 Mar 2010 15:06:05 +0100

Authors: Crystal RG PMID: 20228386 [PubMed - in process] (Source: American Journal of Respiratory Cell and Molecular Biology)

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Antiangiogenic Gene Therapy of Solid Tumor by Systemic Injection of Polyplex Micelles Loading Plasmid DNA Encoding Soluble Flt-1

Molecular Pharmaceutics — Wed, 17 Mar 2010 14:05:41 +0100

Molecular Pharmaceutics, Volume 0, Issue 0, Articles ASAP (As Soon As Publishable). (Source: Molecular Pharmaceutics)

Efficient inhibition of hepatitis B virus replication by hepatitis delta virus ribozymes delivered by targeting retrovirus

BioMed Central — Wed, 17 Mar 2010 00:00:00 +0100

Conclusions: These data suggest that this system provides a new approach for targeting hepatocytes and has a great potential in gene therapy for HBV infection. (Source: BioMed Central)

Pluripotent plasticity of stem cells and liver repopulation

Cell Biochemistry and Function — Tue, 16 Mar 2010 00:00:00 +0100

Different types of stem cells have a role in liver regeneration or fibrous repair during and after several liver diseases. Otherwise, the origin of hepatic and/or extra-hepatic stem cells in reactive liver repopulation is under controversy. The ability of the human body to self-repair and replace the cells and tissues of some organs is often evident. It has been estimated that complete renewal of liver tissue takes place in about a year. Replacement of lost liver tissues is accomplished by proliferation of mature hepatocytes, hepatic oval stem cells differentiation, and sinusoidal cells as support. Hepatic oval cells display a distinct phenotype and have been shown to be a bipotential progenitor of two types of epithelial cells found in the liver, hepatocytes, and bile ductular cells. In g...

JCI online early table of contents: March 15, 2010

EurekAlert! - Cancer — Mon, 15 Mar 2010 04:00:00 +0100

(Journal of Clinical Investigation) This release contains summaries, links to PDFs, and contact information for the following newsworthy papers to be published online, March 15th, 2010, in the JCI: Targeted gene therapy beneficial to mice with spinal muscular atrophy; Sealing the deal to block heart failure in dogs with muscular dystrophy; Cancer drug beneficial in models of infectious disease; Why immune and conventional therapies combine to kill tumors; Possible new drug target for head and neck cancers; and others. (Source: EurekAlert! - Cancer)

Gene Target Beats Oil Remedy

Scientific American - Official RSS Feed — Fri, 12 Mar 2010 14:00:00 +0100

The 1992 tearjerker Lorenzo’s Oil told the true story of one family’s struggle to save their son from X-linked adrenoleukodystrophy (ALD), a deadly degenerative brain disease. Unfortunately, over the ensuing years, the oil of the film’s title, a dietary supplement, has not panned out as the cure many people hoped it would be. Now a paper in the November 2009 issue of Science suggests that the long-sought cure may come from gene therapy--a famously hyped approach to treatment that tragically caused the death of a teenage experimental subject in 1999.Since then, however, researchers have continued to cautiously pursue gene therapy for certain disorders with known genetic origins. ALD, for instance, is caused by mutations in a gene called ABCD1, leading to unusually high lev...

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Gene Target Beats Oil Remedy

Scientific American Topic - Medical Technology — Fri, 12 Mar 2010 14:00:00 +0100

Genetix Pharmaceuticals Secures $35M in Series B Funding

HSMN NewsFeed — Fri, 12 Mar 2010 13:06:13 +0100

Company Focused On Advancing Breakthrough Clinical Programs in Gene Therapy CAMBRIDGE, Mass.--(HSMN NewsFeed)--Genetix Pharmaceuticals, a leading gene therapy company developing breakthrough treatments for severe genetic disorders, announced today that ... Biopharmaceuticals, Venture CapitalGenetix Pharmaceuticals (Source: HSMN NewsFeed)

Medicine's Future Could Lie in Each Patient's Genome

MedlinePlus Health News — Thu, 11 Mar 2010 21:00:00 +0100

In two studies, scientists quickly scanned individuals' DNA to get at causes of disease Source: HealthDay Related MedlinePlus Pages: Genes and Gene Therapy, Genetic Testing (Source: MedlinePlus Health News)

Body's Response to Foods' Smell, Taste Could Be Diabetes Risk Factor

MedlinePlus Health News — Thu, 11 Mar 2010 19:00:00 +0100

A gene mutation could trigger key insulin reaction in some, researchers say Source: HealthDay Related MedlinePlus Pages: Diabetes, Genes and Gene Therapy, Taste and Smell Disorders (Source: MedlinePlus Health News)

Start Of Phase I/II Gene Therapy Clinical Trial For Hemophilia B

Health News from Medical News Today — Thu, 11 Mar 2010 11:00:00 +0100

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced that the first patient has been dosed in the Phase I/II exploratory clinical trial with a gene therapy product for hemophilia B, a seriously debilitating and potentially lethal disease. The trial is an open label dose-escalation study using a vector-gene combination developed at the renowned St. Jude Children's Research Hospital. Dr. Arthur W. Nienhuis of St. Jude is the principal investigator of the on-going trial. The work was initiated at St. Jude more than a decade ago by Drs... (Source: Health News from Medical News Today)

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Start Of Phase I/II Gene Therapy Clinical Trial For Hemophilia B

Genetics News From Medical News Today — Thu, 11 Mar 2010 11:00:00 +0100

How To Fortify The Immunity Of HIV Patients

Health News from Medical News Today — Thu, 11 Mar 2010 09:00:00 +0100

New findings from a UniversitÃ© de MontrÃ©al and the Vaccine and Gene Therapy Institute of Florida (VGTI) study, in collaboration with scientists from the NIH and the McGill University Hospital Center, may soon lead to an expansion of the drug arsenal used to fight HIV. The Canada-U.S. study published today in the journal Nature Medicine characterizes the pivotal role of two molecules, PD-1 and IL-10, in influencing the function of CD4/T-helper cells and altering their ability to fight HIV... (Source: Health News from Medical News Today)

How To Fortify The Immunity Of HIV Patients

Immune System / Vaccines News From Medical News Today — Thu, 11 Mar 2010 09:00:00 +0100

VHL and PTEN loss coordinate to promote mouse liver vascular lesions

Angiogenesis — Thu, 11 Mar 2010 02:45:21 +0100

Abstract Von Hippel-Lindau (VHL) inactivation develops a tumor syndrome characterized by highly vascularized tumors as a result of hypoxia inducible factors (HIF) stabilization. The most common manifestation is the development of hemangioblastomas typically located in the central nervous system and other organs including the liver. PTEN (Phosphatase and tension homologue deleted on chromosome 10) inactivation also upregulates HIF-1α and may take part in promoting vascular lesions in tumors. The coordinate effect of loss of these tumor suppressors on HIF levels, and the subsequent effect on vascular lesion formation would elucidate the potential for mechanisms to modify HIF dosage supplementally and impact tumor phenotype. We therefore employed models of somatic conditional...

Mesenchymal stem cells as therapeutic tools and gene carriers in liver fibrosis and hepatocellular carcinoma

Gene Therapy — Thu, 11 Mar 2010 00:00:00 +0100

Authors: J B Aquino, M F Bolontrade, M G García, O L Podhajcer & G Mazzolini (Source: Gene Therapy)

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Comparative efficacy of dermal fibroblast-mediated and direct adenoviral bone morphogenetic protein-2 gene therapy for bone regeneration in an equine rib model

Gene Therapy — Thu, 11 Mar 2010 00:00:00 +0100

Authors: A Ishihara, L J Zekas, S E Weisbrode & A L Bertone (Source: Gene Therapy)

Articular cartilage repair by genetically modified bone marrow aspirate in sheep

Gene Therapy — Thu, 11 Mar 2010 00:00:00 +0100

Authors: A Ivkovic, A Pascher, D Hudetz, D Maticic, M Jelic, S Dickinson, M Loparic, M Haspl, R Windhager & M Pecina (Source: Gene Therapy)

An ex vivo loop system models the toxicity and efficacy of PEGylated and unmodified adenovirus serotype 5 in whole human blood

Gene Therapy — Thu, 11 Mar 2010 00:00:00 +0100

Authors: A Danielsson, G Elgue, B M Nilsson, B Nilsson, J D Lambris, T H Tötterman, S Kochanek, F Kreppel & M Essand (Source: Gene Therapy)

Oncolytic herpes simplex virus armed with xenogeneic homologue of prostatic acid phosphatase enhances antitumor efficacy in prostate cancer

Gene Therapy — Thu, 11 Mar 2010 00:00:00 +0100

Authors: P Castelo-Branco, B J Passer, J S Buhrman, S Antoszczyk, M Marinelli, C Zaupa, S D Rabkin & R L Martuza (Source: Gene Therapy)

Anticonvulsant effects and behavioural outcomes of rAAV serotype 1 vector-mediated neuropeptide Y overexpression in rat hippocampus

Gene Therapy — Thu, 11 Mar 2010 00:00:00 +0100

Authors: F Noe, V Vaghi, C Balducci, H Fitzsimons, R Bland, D Zardoni, G Sperk, M Carli, M J During & A Vezzani (Source: Gene Therapy)

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Organ-specific shifts in mtDNA heteroplasmy following systemic delivery of a mitochondria-targeted restriction endonuclease

Gene Therapy — Thu, 11 Mar 2010 00:00:00 +0100

Authors: S R Bacman, S L Williams, S Garcia & C T Moraes (Source: Gene Therapy)

Scientists At Institute Of Bioengineering And Nanotechnology Report Research In Cancer Gene Therapy

Cancercompass News: Other Cancer — Thu, 11 Mar 2010 00:00:00 +0100

A new study, 'Combinatorial control of suicide gene expression by tissue-specific promoter and microRNA regulation for cancer therapy,' is now available. Transcriptional targeting using a tissue-specific cellular promoter is proving to be a powerful means for restricting transgene expression in targeted tissues. (Source: Cancercompass News: Other Cancer)

Investigators At University Of Chicago, Brain Tumor Center Have Published New Data On Cancer Gene Therapy

Cancercompass News: Other Cancer — Thu, 11 Mar 2010 00:00:00 +0100

A report, 'Virotherapy against malignant glioma stem cells,' is newly published data in Cancer Letters. Glioblastoma multiforme, the most common primary intracranial malignancy, is associated with very poor outcome despite advances in surgical techniques and chemo-and radiation therapy. Many novel treatment modalities are being investigated with varying amount of success, researchers in the United States report. (Source: Cancercompass News: Other Cancer)

Building Better Chimeric Antigen Receptors for Adoptive T cell Therapy.

Current Gene Therapy — Thu, 11 Mar 2010 00:00:00 +0100

Authors: Bridgeman JS, Hawkins RE, Hombach AA, Abken H, Gilham DE The last few years has seen the transfer of two decades of research into Chimeric Antigen Receptors (CARs) into clinical trials. Despite this extensive research, there is still a great deal of debate into the optimal design strategy for these, primarily, anti-cancer entities. The archetypal CAR consists of a single-chain antibody fragment, specific to a tumour-associated antigen, fused to a component of the T-cell receptor complex (typically CD3zeta) which on antigen binding primes the engrafted T-cell for anti-tumour activity. The modular nature of these artificial receptors has enabled researchers to modify aspects of their structure, including the extracellular spacer, transmembrane and cytoplasmic domain, to achieve ...

Effect of Administration Route on the Biodistribution and Shedding of Replication-Deficient AAV2. A Qualitative Modelling Approach.

Current Gene Therapy — Thu, 11 Mar 2010 00:00:00 +0100

Authors: Brandon EF, Hermsen HP, van Eijkeren JC, Tiesjema B Gene therapy is a rapidly developing field in which recombinant nucleic acid sequences are introduced to individuals to regulate, repair, replace, add or delete a genetic sequence. Recombinant adeno-associated viral (AAV) vectors, especially AAV2, are frequently used in gene therapy. Knowledge on the biodistribution and potential shedding of AAV2 is crucial to evaluate the risks of infection with the viral vector for the patient and the environment. Literature was analysed for biodistribution and shedding data for AAV2. Preclinical and clinical studies were included with a focus on the influence of the administration route on spreading. Based on biodistribution and shedding data, a qualitative model for the biodistribution an...

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Effect of Administration Route on the Biodistribution and Shedding of Replication Deficient HAdV-5. A Qualitative Modelling Approach.

Current Gene Therapy — Thu, 11 Mar 2010 00:00:00 +0100

Authors: Tiesjema B, Hermsen HP, van Eijkeren JC, Brandon EF Centre for Substances and Integrated Risk Assessment, National Institute for Public Health and the Environment, P.O. Box 1, 3720 BA Bilthoven, The Netherlands. esther.brandon@rivm.nl Gene therapy is a rapidly developing field in which recombinant nucleic acid sequences are introduced to individuals. Its therapeutic, prophylactic or diagnostic effect relates directly to the sequence it contains or to the product of genetic expression of this sequence. Recombinant adenoviral vectors (in particular HAdV-5 vectors) are frequently used in gene therapy. Knowledge on biodistribution and shedding is crucial in the risk assessment for the patient and the patient's environment. This review presents a critical overview on biodistributio...

Basic Principles and Clinical Advancements of Muscle Electrotransfer.

Current Gene Therapy — Thu, 11 Mar 2010 00:00:00 +0100

Authors: Hojman P Muscle electrotransfer covers the delivery of molecules to muscle tissue by means of electric pulses. This method has proven highly efficient in transferring, in particular, plasmid DNA to muscles, resulting in long-term expression of the transferred genes. DNA electrotransfer to muscle tissue has clinical potential within DNA vaccination, systemic delivery of therapeutic proteins and correction of gene defects in muscles. In the recent years, DNA electrotransfer to muscle tissue has reached clinical advancement with 8 on-going clinical trials. In the present review, I will draw on the experiences obtained from the clinical studies, in understanding the mechanistic and practical advantages and limits of muscle electrotransfer. The effect of applying electric pulses to...

Entire Family Genome Sequenced for First Time

MedlinePlus Health News — Wed, 10 Mar 2010 22:00:00 +0100

Children receive 30 mutations from each parent, researchers find Source: HealthDay Related MedlinePlus Page: Genes and Gene Therapy (Source: MedlinePlus Health News)

Gene Mutations Identified for Charcot-Marie-Tooth Syndrome

MedlinePlus Health News — Wed, 10 Mar 2010 22:00:00 +0100

Researcher's personal genome used to study neurological disorder Source: HealthDay Related MedlinePlus Pages: Charcot-Marie-Tooth Disease, Genes and Gene Therapy (Source: MedlinePlus Health News)

Therapeutic effects of survivin dominant negative mutant in a mouse model of prostate cancer

Journal of Cancer Research and Clinical Oncology — Wed, 10 Mar 2010 16:10:26 +0100

Conclusions The present study may be of importance in the exploration of the potential application of Lip-mS in the treatment of a broad spectrum of tumors. Content Type Journal ArticleCategory Original PaperDOI 10.1007/s00432-010-0855-2Authors Li Pan, Sichuan University State Key Laboratory of Biotherapy and Cancer Center, West China Hospital, West China Medical School Keyuan Road 4 Chengdu Sichuan People’s Republic of ChinaXing-Chen Peng, Sichuan University State Key Laboratory of Biotherapy and Cancer Center, West China Hospital, West China Medical School Keyuan Road 4 Chengdu Sichuan People’s Republic of ChinaFei Leng, Sichuan University State Key Laboratory of Biotherapy and Cancer Center, West China Hospital, West China Medical School Keyuan Road 4 Chengdu ...

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Antitumor activity of Type I and Type III interferons in BNL hepatoma model

Cancer Immunology, Immunotherapy — Wed, 10 Mar 2010 15:38:27 +0100

Abstract Hepatocellular carcinoma (HCC) occurs most commonly secondary to cirrhosis due to chronic hepatitis C or B virus (HCV/HBV) infections. Type I interferon (IFN-α) treatment of chronic HCV/HBV infections reduces the incidence of HCC in cirrhotic patients. However, IFN-α toxicity limits its tolerability and efficacy highlighting a need for better therapeutic treatments. A recently discovered type III IFN (IFN-λ) has been shown to possess antiviral properties against HCV and HBV in vitro. In phase I clinical trials, IFN-λ treatment did not cause significant adverse reactions. Using a gene therapy approach, we compared the antitumor properties of IFN-α and IFN-λ in a transplantable hepatoma model of HCC. BALB/c mice were inoculated with syngeneic BNL hepatoma cell...

Seventh annual meeting of the Italian Network for Tumor Biotherapy (NIBIT), Siena, 1–3 October 2009

Cancer Immunology, Immunotherapy — Wed, 10 Mar 2010 15:38:23 +0100

Content Type Journal ArticleCategory Meeting ReportDOI 10.1007/s00262-010-0832-2Authors Michele Maio, University Hospital of Siena Division of Medical Oncology and Immunotherapy, Department of Oncology, Istituto Toscano Tumori Strada delle Scotte 14 53100 Siena ItalyHugues J. M. Nicolay, University Hospital of Siena Division of Medical Oncology and Immunotherapy, Department of Oncology, Istituto Toscano Tumori Strada delle Scotte 14 53100 Siena ItalyPaolo A. Ascierto, Istituto Nazionale Tumori “Fondazione Pascale” Naples Melanoma Cooperative Group, Unit of Medical Oncology and Innovative Therapy Naples ItalyFilippo Belardelli, Istituto Superiore di Sanità Department of Cell Biology and Neurosciences Rome ItalyRoberto Camerini, Clinical Research Unit III Sigma Tau SpA Rome ItalyMar...

Center for Cell and Gene Therapy renewed as NIH center

Baylor College of Medicine News — Wed, 10 Mar 2010 05:00:00 +0100

The objective of the PACT program is to advance cellular therapy research in the areas of regeneration of damaged/diseased tissues, organs, … (Source: Baylor College of Medicine News)

Analysis of Human Nucleolar snoRNAs and the Development of snoRNA Modulator of Gene Expression (snoMEN) Vectors.

Mol Biol Cell — Wed, 10 Mar 2010 00:00:00 +0100

Authors: Ono M, Yamada K, Avolio F, Scott MS, van Koningsbruggen S, Barton GJ, Lamond AI Monitoring Editor: A. Gregory Matera Human snoRNAs that copurify with nucleoli isolated from HeLa cells have been characterized. Novel fibrillarin-associated snoRNAs were detected that allowed the creation of a new vector system for the targeted knock-down of one or more genes in mammalian cells. The snoMEN (snoRNA Modulator of gene ExpressioN) vector technology is based on snoRNA HBII-180C, which contains an internal sequence that can be manipulated to make it complementary to RNA targets. Gene specific knock-downs are demonstrated for endogenous cellular proteins and for G/YFP-fusion proteins. Multiplex snoMEN vectors coexpress multiple snoRNAs in one transcript, targeted either to different gene...

Genetic Variant Raises Lung Cancer Risk

MedlinePlus Health News — Tue, 09 Mar 2010 19:00:00 +0100

It doesn't matter whether you smoke a little or a lot, study finds Source: HealthDay Related MedlinePlus Pages: Genes and Gene Therapy, Lung Cancer, Secondhand Smoke (Source: MedlinePlus Health News)

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Fortifying The Immunity Of HIV Patients

HIV / AIDS News From Medical News Today — Tue, 09 Mar 2010 11:00:00 +0100

Fortifying The Immunity Of HIV Patients

Health News from Medical News Today — Tue, 09 Mar 2010 11:00:00 +0100

New findings from a Universite de Montreal and the Vaccine and Gene Therapy Institute of Florida (VGTI) study, in collaboration with scientists from the NIH and the McGill University Health Center, may soon lead to an expansion of the drug arsenal used to fight HIV. The Canada-U.S. study published in the journal Nature Medicine characterizes the pivotal role of two molecules, PD-1 and IL-10, in influencing the function of CD4/T-helper cells and altering their ability to fight HIV... (Source: Health News from Medical News Today)

Lumenal Part of the DC-LAMP Protein Is Not Required for Induction of Antigen-Specific T Cell Responses by Means of Antigen-DC-LAMP Messenger RNA-Electroporated Dendritic Cells

Human Gene Therapy — Tue, 09 Mar 2010 04:02:38 +0100

Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)

New Approach to Immune Cell Analysis Seen as First Step to Better Distinguish Health and Disease

MedlinePlus Health News — Mon, 08 Mar 2010 17:47:38 +0100

Source: National Institute of Allergy and Infectious Diseases - Related MedlinePlus Pages: Genes and Gene Therapy, Immune System and Disorders (Source: MedlinePlus Health News)

Ark's gene therapy stumbles at the finish line

Nature Biotechnology — Mon, 08 Mar 2010 17:42:37 +0100

Nature Biotechnology 28, 183 (2010). doi:10.1038/nbt0310-183 Author: Peter Mitchell (Source: Nature Biotechnology)

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Toward gene therapy of premature ovarian failure: intraovarian injection of adenovirus expressing human FSH receptor restores folliculogenesis in FSHR(-/-) FORKO mice

Molecular Human Reproduction — Mon, 08 Mar 2010 15:51:10 +0100

In conclusion, intra-ovarian injection of an adenovirus expressing human FSHR gene is able to restore FSH responsiveness and reinitiate ovarian folliculogenesis as well as resume estrogen production in female FORKO mice. Ad-LacZ injections indicate the absence of systemic viral dissemination or germ line transmission of adenovirus DNA to offspring. (Source: Molecular Human Reproduction)

piggyBac Transposon/Transposase System to Generate CD19-Specific T Cells for the Treatment of B-Lineage Malignancies

Human Gene Therapy — Mon, 08 Mar 2010 13:20:18 +0100

Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)

Tat-BMPs-PAMAM Conjugates Enhance Therapeutic Effect of Small Interference RNA on U251 Glioma Cells in Vitro and in Vivo

Human Gene Therapy — Mon, 08 Mar 2010 13:07:21 +0100

Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)

Gene Variant May Help Some Overcome Adversity

MedlinePlus Health News — Sun, 07 Mar 2010 14:00:00 +0100

It appears to disconnect the link between stress and death risk, researchers say Source: HealthDay Related MedlinePlus Pages: Genes and Gene Therapy, Mental Health, Stress (Source: MedlinePlus Health News)

Scientists Still Hopeful About Gene Therapy's Promise

NPR Health and Science — Sun, 07 Mar 2010 05:00:00 +0100

For 25 years, scientists have touted the promise of gene therapy to treat human diseases, but only a handful of therapies have shown progress. Nonetheless, proponents remain optimistic and say the approach may yet revolutionize medicine.» E-Mail This » Add to Del.icio.us (Source: NPR Health and Science)

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How to fortify the immunity of HIV patients

EurekAlert! - Medicine and Health — Sun, 07 Mar 2010 05:00:00 +0100

(University of Montreal) New findings from a Université de Montréal and the Vaccine and Gene Therapy Institute of Florida study, in collaboration with scientists from the NIH and the McGill University Health Center, may soon lead to an expansion of the drug arsenal used to fight HIV. The Canada-US study is published today in the journal Nature Medicine. (Source: EurekAlert! - Medicine and Health)

Blocking effects of siRNA on VEGF expression in human colorectal cancer cells.

World Journal of Gastroenterology : WJG — Sun, 07 Mar 2010 00:00:00 +0100

CONCLUSION: VEGF, FLT-1 and FLK-1 are associated with colorectal carcinogenesis. siRNA silencing of the VEGF gene suppresses proliferation, and induces apoptosis in HCT116 cells. The results suggest that VEGF may be a new gene therapy target for colorectal cancer. PMID: 20205278 [PubMed - in process] (Source: World Journal of Gastroenterology : WJG)

Selective Suicide Gene Therapy of Colon Cancer Exploiting the Urokinase Plasminogen Activator Receptor Promoter

BioDrugs — Sat, 06 Mar 2010 14:07:50 +0100

(Source: BioDrugs)

Health headlines: Binge drinking, Wii workout games and CPR

Thrive, Children's Hospital Boston — Sat, 06 Mar 2010 13:00:37 +0100

Other stories we’ve been reading: Read one father’s story on how he became an advocate for safer teen driving. Check out these safe driving tips for your teen. [Read about the dangers of drowsy driving.] Advertising guilt doesn’t curb binge drinking. Teen alcohol and marijuana use is on the rise. [A recent teen drug survey predicted this.] Young people who smoke marijuana for long periods of time are more likely to risk psychosis. Despite studies proving otherwise, some parents still believe that vaccines cause autism. Gene therapy is closer to restoring eyesight to blind patients. [Read about how a novel surgery saves one baby’s vision.] A blood test can help sort out milk allergies. [Watch Brett go through a milk exposure desensitization trial.] Screening athletes could prevent s...

Functional aspects of factor VIII expression after transplantation of genetically-modified hematopoietic stem cells for hemophilia A

The Journal of Gene Medicine — Fri, 05 Mar 2010 00:00:00 +0100

Major complications with respect to the development of gene therapy treatments for hemophilia A include low factor VIII (fVIII) expression and humoral immune responses resulting in inhibitory anti-fVIII antibodies. We previously achieved sustained curative fVIII activity levels in hemophilia A mice after nonmyeloablative transplantation of genetically-modified hematopoietic stem cells (HSCs) encoding a B-domain deleted porcine fVIII (BDDpfVIII) transgene with no evidence of an immune response.Mouse HSCs were transduced using MSCV-based recombinant virus encoding BDDpfVIII and transplanted into hemophilia A mice. Transplanted mice were followed for donor cell engraftment, fVIII expression and activity, and generation of anti-fVIII immune response.We now show that: (i) the protein expressed ...

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pFARs, Plasmids free of antibiotic resistance markers display high-level transgene expression in muscle, skin and tumour cells

The Journal of Gene Medicine — Fri, 05 Mar 2010 00:00:00 +0100

Nonviral gene therapy requires a high yield and a low cost production of eukaryotic expression vectors that meet defined criteria such as biosafety and quality of pharmaceutical grade. To fulfil these objectives, we designed a novel antibiotic-free selection system.The proposed strategy relies on the suppression of a chromosomal amber mutation by a plasmid-borne function. We first introduced a nonsense mutation into the essential Escherichia coli thyA gene, resulting in thymidine auxotrophy. The bacterial strain was optimized for the production of small and novel plasmids free of antibiotic resistance markers (pFARs) and encoding an amber suppressor t-RNA. Finally, the potentiality of pFARs as eukaryotic expression vectors was assessed by monitoring luciferase activities after electrotrans...

Radiation-inducible silencing of uPA and uPAR in vitro and in vivo in meningioma.

International Journal of Oncology — Thu, 04 Mar 2010 22:59:35 +0100

Authors: Rao Gogineni V, Kumar Nalla A, Gupta R, Gorantla B, Gujrati M, Dinh DH, Rao JS Stereospecific radiation treatment offers a distinct opportunity for temporal and spatial regulation of gene expression at tumor sites by means of inducible promoters. To this end, a plasmid, pCArG-U2, was constructed by incorporating nine CArG elements (in tandem) of EGR1 gene upstream to uPA and uPAR siRNA oligonucleotides in a pCi-neo vector. Radiation-induced siRNA expression was detected in a meningioma cell line (IOMM-Lee). Immunoblotting and RT-PCR analyses confirmed downregulation of uPA and uPAR. A similar effect was observed in transfected cells followed by H2O2 treatment. Moreover, pre-treatment of transfected cells with N-acetyl L-cysteine blocked the silencing of uPA and uPAR, which fur...

A new system for regulated functional gene expression for gene therapy applications: Nuclear delivery of a p16INK4A-estrogen receptor carboxy terminal fusion protein only in the presence of estrogen.

International Journal of Oncology — Thu, 04 Mar 2010 22:58:57 +0100

Authors: Tamura T, Kanuma T, Nakazato T, Faried LS, Aoki H, Minegishi T The clinical use of gene therapy requires tight regulation of the gene of interest and functional expression only when it is needed. Thus, it is necessary to develop ways of regulating functional gene expression with exogenous stimuli. Many regulatable systems are currently under development. For example, the tetracycline-dependent transcriptional switch has been successfully employed for in vivo preclinical applications. However, there are no examples of regulatable systems that have been employed in human clinical trials. In the present study, we established an adenovirus-delivered functional gene expression system that is regulated by estrogen. This system uses p16INK4A fused at its C-terminus to the ligand-bind...

Second dose of gene therapy for inherited blindness proves safe in animal studies

ScienceDaily Headlines — Thu, 04 Mar 2010 17:31:32 +0100

A research team that conducted the gene therapy trial for an inherited blindness reports that a study in animals has shown that a second injection of genes into the opposite, previously untreated eye is safe and effective, with no signs of interference from unwanted immune reactions following the earlier injection. These new findings suggest that patients who benefit from gene therapy in one eye may experience similar benefits from treatment in the other eye. (Source: ScienceDaily Headlines)

Second Dose Of Gene Therapy For Inherited Blindness Proves Safe In Animal Study

Eye Health / Optometry News From Medical News Today — Thu, 04 Mar 2010 12:00:00 +0100

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Second Dose Of Gene Therapy For Inherited Blindness Proves Safe In Animal Study

Health News from Medical News Today — Thu, 04 Mar 2010 12:00:00 +0100

Gene therapy for a severe inherited blindness, which produced dramatic improvements last year in 12 children and young adults who received the treatment in a clinical trial, has cleared another hurdle. The same research team that conducted the human trial now reports that a study in animals has shown that a second injection of genes into the opposite, previously untreated eye is safe and effective, with no signs of interference from unwanted immune reactions following the earlier injection... (Source: Health News from Medical News Today)

A dual promoter lentiviral vector for the in vivo evaluation of gene therapeutic approaches to axon regeneration after spinal cord injury

Gene Therapy — Thu, 04 Mar 2010 00:00:00 +0100

Authors: K Löw, A Blesch, J Herrmann & M H Tuszynski (Source: Gene Therapy)

Adeno-associated virus-mediated delivery of kringle 5 of human plasminogen inhibits orthotopic growth of ovarian cancer

Gene Therapy — Thu, 04 Mar 2010 00:00:00 +0100

Authors: T M B Nguyen, I V Subramanian, X Xiao, P Nguyen & S Ramakrishnan (Source: Gene Therapy)

Gene-modified T cells as immunotherapy for multiple myeloma and acute myeloid leukemia expressing the Lewis Y antigen

Gene Therapy — Thu, 04 Mar 2010 00:00:00 +0100

Authors: S Peinert, H M Prince, P M Guru, M H Kershaw, M J Smyth, J A Trapani, P Gambell, S Harrison, A M Scott, F E Smyth, P K Darcy, K Tainton, P Neeson, D S Ritchie & D Hönemann (Source: Gene Therapy)

Cutaneous vaccination using microneedles coated with hepatitis C DNA vaccine

Gene Therapy — Thu, 04 Mar 2010 00:00:00 +0100

Authors: H S Gill, J Söderholm, M R Prausnitz & M Sällberg (Source: Gene Therapy)

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Progress and prospects: nuclear import of nonviral vectors

Gene Therapy — Thu, 04 Mar 2010 00:00:00 +0100

Authors: A P Lam & D A Dean (Source: Gene Therapy)

Intraarticular Treatments for Osteoarthritis: New Perspectives.

Current Drug Targets — Thu, 04 Mar 2010 00:00:00 +0100

Authors: Chevalier X To date, no targeted treatments for osteoarthritis have been developed. Therefore the challenge for the next years is to find a treatment that may slow down the progression of the disease. Osteoarthritis of the weight-bearing joints, such as knee OA, is more a local mechanical driven disease than a generalized one. To reach a non-vascularised tissue such as the cartilage, local intra-articular administration of drugs should be considered. The purpose of this review is to evaluate the advantages of local intra-articular drug administration compared with a systemic one in patients with osteoarthritis of weight-bearing joints. New perspectives of such strategy are reviewed, including anti-cytokine therapy, gene therapy, delivery of growth factors, stem cells therapy a...

The Biology of the Sodium Iodide Symporter and its Potential for Targeted Gene Delivery.

Current Cancer Drug Targets — Thu, 04 Mar 2010 00:00:00 +0100

Authors: Hingorani M, Spitzweg C, Vassaux G, Newbold K, Melcher A, Pandha H, Vile R, Harrington K The sodium iodide symporter (NIS) is responsible for thyroidal, salivary, gastric, intestinal and mammary iodide uptake. It was first cloned from the rat in 1996 and shortly thereafter from human and mouse tissue. In the intervening years, we have learned a great deal about the biology of NIS. Detailed knowledge of its genomic structure, transcriptional and post-transcriptional regulation and pharmacological modulation has underpinned the selection of NIS as an exciting approach for targeted gene delivery. A number of in vitro and in vivo studies have demonstrated the potential of using NIS gene therapy as a means of delivering highly conformal radiation doses selectively to tumours. This ...

Molecular Evolution of Theta Class Glutathione Transferase for Enhanced Activity with the Anticancer Drug 1,3-Bis-(2-Chloroethyl)-1-Nitrosourea and Other Alkylating Agents.

Archives of Biochemistry and Biophysics — Thu, 04 Mar 2010 00:00:00 +0100

Authors: Larsson AK, Shokeer A, Mannervik B Glutathione transferase (GST) displaying enhanced activity with the cytostatic drug 1,3-bis-(2-chloroethyl)-1-nitrosourea (BCNU) and structurally related alkylating agents was obtained by molecular evolution. Mutant libraries created by recursive recombination of cDNA coding for human and rodent Theta class GSTs were heterologously expressed in Escherichia coli and screened with the surrogate substrate 4-nitrophenethylbromide (NPB) for enhanced alkyltransferase activity. A mutant with a 70-fold increased catalytic efficiency with NPB, compared to human GST T1-1, was isolated. The efficiency in degrading BCNU had improved 170-fold, significantly more than with the model substrate NPB. The enhanced catalytic activity of the mutant GST was also ...

Links Seen Between Gut Bacteria and Disease

MedlinePlus Health News — Wed, 03 Mar 2010 18:08:38 +0100

Some of the hundreds of bacteria found in the digestive systems of humans may be linked to specific diseases like cancer, diabetes and obesity, an international team of scientists said in a paper on Thursday.Source: Reuters Health Related MedlinePlus Page: Genes and Gene Therapy (Source: MedlinePlus Health News)

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Gene Therapy to Improve High-Density Lipoprotein Metabolism and Function.

Current Pharmaceutical Design — Wed, 03 Mar 2010 00:00:00 +0100

Authors: Van Craeyveld E, Gordts S, Feng Y, Jacobs F, De Geest B Plasma levels of high-density lipoprotein (HDL) cholesterol and its major apolipoprotein (apo), apo A-I, are inversely correlated with the incidence of ischemic cardiovascular diseases. Till now, evaluation of the hypothesis that elevation of HDL cholesterol reduces atherosclerotic burden and/or decreases ischemic cardiovascular events in humans has been hampered by the lack of drugs that selectively increase HDL cholesterol. In contrast to the lack of clinical data, evidence for a direct causal role of HDL in modulating atherogenesis in experimental models has been provided by investigations in human apo A-I transgenic mice and rabbits. The development of gene transfer technologies with a sufficiently high therapeutic in...

Quantification of cellular uptake and in vivo tracking of transduction using real-time monitoring.

Biochemical and Biophysical Research communications — Wed, 03 Mar 2010 00:00:00 +0100

Authors: Lee MS, Kwon EH, Choi HS, Kwon SH, Shim IS, Lee SK, Her S Protein transduction domains (PTDs) are short amino acid sequences that promote their own translocation across the cell plasma membrane and have been studied for possible use in drug delivery and gene therapy. However, no direct method to quantify transduction is available. Here, using a new luciferase-tagged human PTD, we show that cellular uptake levels can be determined in a reliable manner. Furthermore, we show that enhanced in vivo tracking by human PTD can be quantified in a mouse model. This is the first report on the direct quantification of PTD transduction in vitro and in vivo, which will be necessary for studying its possible therapeutic application in drug delivery and gene therapy. PMID: 20206600 [PubMe...

Site-specific gene therapy for cardiovascular disease

Current Opinion in Drug Discovery & Development — Wed, 03 Mar 2010 00:00:00 +0100

No description available (Source: Current Opinion in Drug Discovery & Development)

Cluster of Genes Tied to Alcoholism

MedlinePlus Health News — Tue, 02 Mar 2010 22:00:00 +0100

Finding part of ongoing research to determine how they affect risk in people Source: HealthDay Related MedlinePlus Pages: Alcoholism, Genes and Gene Therapy (Source: MedlinePlus Health News)

Gene Therapy Reverses Effects Of Lethal Childhood Muscle Disorder In Mice

Pediatrics News From Medical News Today — Tue, 02 Mar 2010 12:00:00 +0100

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Gene Therapy Reverses Effects Of Lethal Childhood Muscle Disorder In Mice

Health News from Medical News Today — Tue, 02 Mar 2010 12:00:00 +0100

Reversing a protein deficiency through gene therapy can correct motor function, restore nerve signals and improve survival in mice that serve as a model for the lethal childhood disorder spinal muscular atrophy, new research shows. This muscle-wasting disease results when a child's motor neurons - nerve cells that send signals from the spinal cord to muscles - produce insufficient amounts of what is called survival motor neuron protein, or SMN... (Source: Health News from Medical News Today)

Therapeutic Endoscopic Ultrasound for Biliary and Pancreatic Disorders

Current Gastroenterology Reports — Tue, 02 Mar 2010 10:08:00 +0100

This article reviews existing data and focuses on established and emerging EUS techniques for accessing and draining the bile and pancreatic ducts. Content Type Journal ArticleDOI 10.1007/s11894-010-0090-7Authors Michael J. Levy, Mayo Clinic College of Medicine Division of Gastroenterology and Hepatology 200 First Street SW, Charlton 8 Rochester MN 55905 USA Journal Current Gastroenterology ReportsOnline ISSN 1534-312XPrint ISSN 1522-8037 (Source: Current Gastroenterology Reports)

Cationic copolymers nanoparticles for nonviral gene vectors: Synthesis, characterization, and application in gene delivery.

Cell Research — Tue, 02 Mar 2010 00:00:00 +0100

Authors: d'Ayala GG, Calarco A, Malinconico M, Laurienzo P, Petillo O, Torpedine A, Peluso G The major aim of nonviral delivery systems for gene therapy is to mediate high levels of gene expression with low toxicity. Nowadays, one of the most successful synthetic polycations used in gene delivery research is poly(ethylenimine) (PEI) in its high-molecular weight (HMW) branched form. However, PEI is not the ideal transfection agent in vivo because of its overwhelming cytotoxicity. To overcome its toxic effects with a minimal impact on transfection efficiency, PEI has been conjugated with several nonionic biocompatible polymers. Here, we describe the synthesis of nanosized particles consisting of HMW PEI (25 kDa) crosslinked with poly(epsilon-caprolactone) (PCL, 50-60 kDa), a biodegradabl...

Gene therapy reverses effects of lethal childhood muscle disorder in mice

ScienceDaily Headlines — Mon, 01 Mar 2010 16:00:00 +0100

Reversing a protein deficiency through gene therapy can correct motor function, restore nerve signals and improve survival in mice that serve as a model for the lethal childhood disorder spinal muscular atrophy, new research shows. This muscle-wasting disease results when a child's motor neurons -- nerve cells that send signals from the spinal cord to muscles -- produce insufficient amounts of what is called survival motor neuron protein, or SMN. (Source: ScienceDaily Headlines)

AAV-2: how can the capsid be modified to improve the viral vector in gene therapy?

Future Virology — Mon, 01 Mar 2010 15:56:13 +0100

Future Virology , March 2010, Vol. 5, No. 2, Pages 133-135. (Source: Future Virology)

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Challenges with advanced therapy medicinal products and how to meet them

Nature Reviews Drug Discovery — Mon, 01 Mar 2010 14:48:42 +0100

Authors: Advanced therapy medicinal products (ATMPs), which include gene therapy medicinal products, somatic cell therapy medicinal products and tissue-engineered products, are at the cutting edge of innovation and offer a major hope for various diseases for which there are limited or no therapeutic options. They have (Source: Nature Reviews Drug Discovery)

Deal watch: Novartis licenses GenVec's hearing loss programme

Nature Reviews Drug Discovery — Mon, 01 Mar 2010 14:48:42 +0100

Deal watch: Novartis licenses GenVec's hearing loss programme Nature Reviews Drug Discovery 9, 182 (2010). doi:10.1038/nrd3126 Novartis has acquired exclusive global rights to GenVec's preclinical hearing loss and balance disorders programme. The programme has been investigating gene therapy to express atonal homologue 1 (ATOH1) in the inner ear to regenerate the sensory hair cells involved in detecting sound and (Source: Nature Reviews Drug Discovery)

A Placental Growth Factor Variant Unable to Recognize Vascular Endothelial Growth Factor (VEGF) Receptor-1 Inhibits VEGF-Dependent Tumor Angiogenesis via Heterodimerization

Cancer Research — Mon, 01 Mar 2010 05:07:37 +0100

A mechanistically unique approach to target VEGF-mediated angiogenesis via gene therapy approach is presented. (Source: Cancer Research)

Selective Suicide Gene Therapy of Colon Cancer Cell Lines Exploiting Fibroblast Growth Factor 18 Promoter

Cancer Biotherapy and Radiopharmaceuticals — Mon, 01 Mar 2010 04:54:18 +0100

Cancer Biotherapy & Radiopharmaceuticals Feb 2010, Vol. 25, No. 1: 105-116. (Source: Cancer Biotherapy and Radiopharmaceuticals)

Muscle Gene Therapy

Springer Biomedical Sciences titles — Mon, 01 Mar 2010 00:13:26 +0100

(Source: Springer Biomedical Sciences titles)

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Thioredoxin-1 Gene Therapy Enhances Angiogenic Signaling and Reduces Ventricular Remodeling in Infarcted Myocardium of Diabetic Rats.

Circulation — Mon, 01 Mar 2010 00:00:00 +0100

Conclusions-This study demonstrates for the first time that impairment of angiogenesis and myocardial dysfunction can be regulated by Ad.Trx1 gene therapy in streptozotocin-induced diabetic rats subjected to infarction. PMID: 20194885 [PubMed - as supplied by publisher] (Source: Circulation)

Gene Links to Celiac Disease May Help Drug Search

MedlinePlus Health News — Sun, 28 Feb 2010 18:02:35 +0100

Scientists have identified new genetic links to celiac disease and say their findings could speed the search for better ways to diagnose and treat the gluten-intolerance disorder.Source: Reuters Health Related MedlinePlus Pages: Celiac Disease, Genes and Gene Therapy (Source: MedlinePlus Health News)

Study: Gene therapy reverses effects of lethal childhood muscle disorder in mice

EurekAlert! - Medicine and Health — Sun, 28 Feb 2010 05:00:00 +0100

(Ohio State University) Reversing a protein deficiency through gene therapy can correct motor function, restore nerve signals and improve survival in mice that serve as a model for the lethal childhood disorder spinal muscular atrophy, new research shows. This muscle-wasting disease results when a child's motor neurons -- nerve cells that send signals from the spinal cord to muscles -- produce insufficient amounts of what is called survival motor neuron protein, or SMN. (Source: EurekAlert! - Medicine and Health)

Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

Nature Biotechnology — Sun, 28 Feb 2010 00:00:00 +0100

Authors: Kevin D Foust, Xueyong Wang, Vicki L McGovern, Lyndsey Braun, Adam K Bevan, Amanda M Haidet, Thanh T Le, Pablo R Morales, Mark M Rich, Arthur H M Burghes & Brian K Kaspar Spinal muscular atrophy (SMA), the most common autosomal recessive neurodegenerative disease affecting children, results in impaired motor neuron function. Despite knowledge of the pathogenic role of decreased survival motor neuron (SMN) protein levels, efforts to increase SMN have not resulted in a treatment for patients. We recently demonstrated that self-complementary adeno-associated virus 9 (scAAV9) can infect ∼60% of motor neurons when injected intravenously into neonatal mice. Here we use scAAV9-mediated postnatal day 1 vascular gene delivery to replace SMN in SMA pups and rescue motor function, ...

Anti-tumor immune response correlates with neurological symptoms in a dog with spontaneous astrocytoma treated by gene and vaccine therapy.

Vaccine — Sat, 27 Feb 2010 00:00:00 +0100

Authors: Pluhar GE, Grogan PG, Seiler C, Goulart M, Santacruz KS, Carlson C, Chen W, Olin MR, Lowenstein PR, Castro MG, Haines SJ, Ohlfest JR Gene therapy and vaccination have been tested in malignant glioma patients with modest, albeit encouraging results. The combination of these therapies has demonstrated synergistic efficacy in murine models but has not been reported in large animals. Gemistocytic astrocytoma (GemA) is a low-grade glioma that typically progresses to lethal malignancy despite conventional therapies. Until now there has been no useful animal model of GemA. Here we report the treatment of a dog with spontaneous GemA using the combination of surgery, intracavitary adenoviral interferon gamma (IFNgamma) gene transfer, and vaccination with glioma cell lysates mixed with ...

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New Treatment Eyed for Deadly Pancreatic Cancer

MedlinePlus Health News — Fri, 26 Feb 2010 22:00:00 +0100

Drug already approved for arthritis looks promising in lab tests, researchers say Source: HealthDay Related MedlinePlus Pages: Genes and Gene Therapy, Pancreatic Cancer (Source: MedlinePlus Health News)

Genes Behind Tooth Development Discovered

MedlinePlus Health News — Fri, 26 Feb 2010 14:00:00 +0100

Scientists say they affect appearance of first one, number of teeth in first year Source: HealthDay Related MedlinePlus Pages: Child Dental Health, Genes and Gene Therapy (Source: MedlinePlus Health News)

An HDAC inhibitor increases AcMNPV gene expression in mammalian cells

Archives of Virology — Fri, 26 Feb 2010 06:45:22 +0100

Abstract The baculovirus Autographa californica multiple nucleopolyhedrovirus (AcMNPV) is used as a safer viral vector in mammalian cells with potential applications in gene therapy. However, the mechanism for the insusceptibility of mammalian cells to proliferative infection by entomopathogenic viruses is not well understood. Here, we studied the significance of epigenetic modifications such as histone acetylation, histone methylation and HP1 accumulation for AcMNPV gene expression in mammalian BHK cells. Real-time PCR and chromatin immunoprecipitation with sodium butyrate revealed an important relationship between viral gene expression and histone acetylation, with implications for a mechanism of suppression of AcMNPV gene expression in BHK cells. Content Type Journa...

Inhibition of Cell Proliferation of Tenon's Capsule Fibroblast by S-Phase Kinase-Interacting Protein 2 Targeting SiRNA through Increasing p27 Protein Level [Glaucoma]

Investigative Ophthalmology — Fri, 26 Feb 2010 01:05:09 +0100

Skp2 siRNA inhibited cell proliferation and decreased cell viability of rTF in vivo and in vitro. SiRNA-mediated gene silencing of Skp2 can be a novel gene therapy to treat scarring after glaucoma surgery by the suppression of p27kip1 downregulation. (Source: Investigative Ophthalmology)

Enhanced anti-tumor effects of combined MDR1 RNA interference and human sodium/iodide symporter (NIS) radioiodine gene therapy using an adenoviral system in a colon cancer model

Cancer Gene Therapy — Fri, 26 Feb 2010 00:00:00 +0100

Enhanced anti-tumor effects of combined MDR1 RNA interference and human sodium/iodide symporter (NIS) radioiodine gene therapy using an adenoviral system in a colon cancer model Cancer Gene Therapy advance online publication, February 26, 2010. doi:10.1038/cgt.2010.3 Authors: S J Ahn, Y H Jeon, Y J Lee, Y L Lee, S-W Lee, B-C Ahn, J-H Ha & J Lee (Source: Cancer Gene Therapy)

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Effective immunotherapy of weakly immunogenic solid tumours using a combined immunogene therapy and regulatory T-cell inactivation

Cancer Gene Therapy — Fri, 26 Feb 2010 00:00:00 +0100

Authors: M C Whelan, G Casey, M MacConmara, J A Lederer, D Soden, J K Collins, M Tangney & G C O'Sullivan (Source: Cancer Gene Therapy)

Reported at CROI: Early Development of Gene Therapy for HIV Promising

Fri, 26 Feb 2010 00:00:00 +0100

“Existing AIDS drugs allow many patients to live fairly normal lives despite being infected with HIV. But they can cause a variety of side effects, and some patients become immune to them over time. … “In [a] new study, the [University of] Pennsylvania [research] team tested a gene therapy approach in which scientists first remove immune cells from patients, tinker with their genes and then put them back into the bodies of the patients. “Eight HIV-infected people took part in the study. After the genetically modified cells were placed back into the patients, ‘we stopped HIV treatment and tried to see what happened,’ [Dr. Pablo] Tebas said. … “The levels of HIV fell below the expected levels in seven of the eight patients, the team found. … “It's still early in...

Gene Mutation in Mice Sheds Light on Autism

MedlinePlus Health News — Thu, 25 Feb 2010 21:00:00 +0100

Drugs targeting obsessive behavior might hold promise for people Source: HealthDay Related MedlinePlus Pages: Autism, Genes and Gene Therapy (Source: MedlinePlus Health News)

A highly efficient short hairpin RNA potently down-regulates CCR5 expression in systemic lymphoid organs in the hu-BLT mouse model

Blood — Thu, 25 Feb 2010 17:22:40 +0100

Inhibiting the expression of the HIV-1 coreceptor CCR5 holds great promise for controlling HIV-1 infection in patients. Here we report stable knockdown of human CCR5 by a short hairpin RNA (shRNA) in a humanized bone marrow/liver/thymus (BLT) mouse model. We delivered a potent shRNA against CCR5 into human fetal liver-derived CD34+ hematopoietic progenitor/stem cells (HPSCs) by lentiviral vector transduction. We transplanted vector-transduced HPSCs solidified with Matrigel and a thymus segment under the mouse kidney capsule. Vector-transduced autologous CD34+ cells were subsequently injected in the irradiated mouse, intended to create systemic reconstitution. CCR5 expression was down-regulated in human T cells and monocytes/macrophages in systemic lymphoid tissues, including gut-associated...

Nonviral Intercellular Adhesion Molecule-1 Small Interfering Ribonucleic Acid Sequences Transfection In Vivo: How Ultrasound Bursts Into Therapy⁎

Journal of the American College of Cardiology — Thu, 25 Feb 2010 13:51:49 +0100

A decade ago, gene therapy of atherosclerosis was thought to have a bright future for its proven capacity to force expression of a protein that is either lacking but necessary (such as the low-density lipoprotein receptor in hyperlipoproteinemia type IIa) or a disease-modifying molecular switch (such as hypoxia-inducible factor 1α in the case of chronic ischemia). Both approaches have been successfully realized experimentally (), but still await clinical approval because of side effects or lack of efficacy. Conceptually, an even more complex attempt is to get rid of an unwanted protein, for example, a pro-inflammatory protein that is leading to intima proliferation and vascular stenosis. In addition to overexpression of dominant negative mutants competing with the functional target protei...

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Ten days to save hearing after deafening sound

New Scientist - Health — Thu, 25 Feb 2010 12:01:56 +0100

A shot of gene therapy was enough to restore dying hair cells in guinea pigs – if administered soon enough after the damage (Source: New Scientist - Health)

A First Step in Viral Gene Therapy for Muscular Dystrophy

Current Neurology and Neuroscience Reports — Thu, 25 Feb 2010 08:02:34 +0100

Content Type Journal ArticleCategory Clinical Trial ReportDOI 10.1007/s11910-010-0090-xAuthors Michio Hirano, Columbia University Medical Center 630 West 168th Street, P&S 4-423 New York NY 10032 USA Journal Current Neurology and Neuroscience ReportsOnline ISSN 1534-6293Print ISSN 1528-4042 (Source: Current Neurology and Neuroscience Reports)

Studies on protective effects of human paraoxonases 1 and 3 on atherosclerosis in apolipoprotein E knockout mice

Gene Therapy — Thu, 25 Feb 2010 00:00:00 +0100

Authors: C Zhang, W Peng, M Wang, J Zhu, Y Zang, W Shi, J Zhang & J Qin (Source: Gene Therapy)

Insulin expressed from endogenously active glucose-responsive EGR1 promoter in bone marrow mesenchymal stromal cells as diabetes therapy

Gene Therapy — Thu, 25 Feb 2010 00:00:00 +0100

Authors: N K F Chen, S Y Tan, G Udolph & O L Kon (Source: Gene Therapy)

TRAIL gene-armed oncolytic poxvirus and oxaliplatin can work synergistically against colorectal cancer

Gene Therapy — Thu, 25 Feb 2010 00:00:00 +0100

Authors: M F Ziauddin, Z S Guo, M E O'Malley, F Austin, P J Popovic, M A Kavanagh, J Li, M Sathaiah, P Thirunavukarasu, B Fang, Y J Lee & D L Bartlett (Source: Gene Therapy)

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Anti-inflammatory effect by lentiviral-mediated overexpression of IL-10 or IL-1 receptor antagonist in rat glial cells and macrophages

Gene Therapy — Thu, 25 Feb 2010 00:00:00 +0100

Authors: M E van Strien, D Mercier, B Drukarch, J J P Brevé, S Poole, R Binnekade, J G J M Bol, B Blits, J Verhaagen & A-M van Dam (Source: Gene Therapy)

Functional recovery of diabetic mouse hearts by glutaredoxin-1 gene therapy: role of Akt-FoxO-signaling network

Gene Therapy — Thu, 25 Feb 2010 00:00:00 +0100

Authors: I Lekli, S Mukherjee, D Ray, N Gurusamy, Y H Kim, A Tosaki, R M Engelman, Y-S Ho & D K Das (Source: Gene Therapy)

Adeno-associated virus type 5-mediated intraarticular administration of tumor necrosis factor small interfering RNA improves collagen-induced arthritis

Arthritis and Rheumatism — Thu, 25 Feb 2010 00:00:00 +0100

RNA interference (RNAi) is a powerful tool for sequence-specific gene silencing, and interest in its application in human diseases is growing. Given the success of recent strategies for administering gene therapy in rheumatoid arthritis using recombinant vectors such as adeno-associated virus type 5 (rAAV5) for optimized intraarticular gene transfer, we undertook the present study to determine the feasibility of using rAAV5-mediated RNAi-based therapy in arthritis.We developed rAAV5 vectors expressing short hairpin small interfering RNA (shRNA) against tumor necrosis factor [alpha] (TNF[alpha]) under H1 promoter, and carrying the enhanced green fluorescent protein (eGFP) reporter gene under cytomegalovirus promoter (rAAV5-shTNF). TNF[alpha] gene silencing was validated in vitro with mouse ...

Soluble Flt-1 gene therapy ameliorates albuminuria but accelerates tubulointerstitial injury in diabetic mice

AJP: Renal Physiology — Wed, 24 Feb 2010 22:28:42 +0100

In conclusion, gene therapy with sFlt-1-AAV1 protects podocytes but accelerates tubulointerstitial injury in diabetic db/db mice. These data suggest systemic overexpression of sFlt-1 will not likely be useful for treating diabetic nephropathy. (Source: AJP: Renal Physiology)

New Round Of UREP Grants For WCMC-Q Students

Health News from Medical News Today — Wed, 24 Feb 2010 09:00:00 +0100

Twenty-eight WCMC-Q students have received grants totaling $280,000 in the latest, seventh, round of funding from the Undergraduate Research Experience Program (UREP) to conduct advanced research projects with faculty members. The students will investigate 11 different topics related to cancer treatment and diagnosis, diabetes care, science learning, purifying drinking water, infectious diseases, the function of specific genes, gene therapy for diseases and the growth and development of children in Qatar. The grants are part of $1... (Source: Health News from Medical News Today)

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TIMP-3 shows potential as gene therapy for prostate cancer

MedWire News - Oncology — Wed, 24 Feb 2010 00:00:00 +0100

Researchers have discovered a potential new gene therapy for prostate cancer in the form of tissue inhibitor of metalloproteinase-3. (Source: MedWire News - Oncology)

Update on the hyper immunoglobulin M syndromes

British Journal of Haematology — Wed, 24 Feb 2010 00:00:00 +0100

The Hyper-immunoglobulin M syndromes (HIGM) are a heterogeneous group of genetic disorders resulting in defects of immunoglobulin class switch recombination (CSR), with or without defects of somatic hypermutation (SHM). They can be classified as defects of signalling through CD40 causing both a humoral immunodeficiency and a susceptibility to opportunistic infections, or intrinsic defects in B cells of the mechanism of CSR resulting in a pure humoral immunodeficiency. A HIGM picture can also be seen as part of generalized defects of DNA repair and in antibody deficiency syndromes, such as common variable immunodeficiency. CD40 signalling defects may require corrective therapy with bone marrow transplantation. Gene therapy, a potential curative approach in the future, currently remains a di...

TIMP-3 shows potential as gene therapy for prostate cancer

MedWire News - Prostate Cancer — Wed, 24 Feb 2010 00:00:00 +0100

Researchers have discovered a potential new gene therapy for prostate cancer in the form of tissue inhibitor of metalloproteinase-3. (Source: MedWire News - Prostate Cancer)

Co-expression of insulin-like growth factor-1 and interleukin-4 in an in vitro inflammatory model.

Cytokine — Wed, 24 Feb 2010 00:00:00 +0100

In this study, we analyze the expression of IGF-1 and IL-4 from a single plasmid vector, where each gene is expressed through an independent promoter and enhancer sequence. Regenerative and anti-inflammatory effects of IGF-1 alone and of both IGF-1 and IL-4 were analyzed in an in vitro chondrocyte inflammatory model. Co-expression of both transgenes in primary chondrocytes was ascertained by immunoassays. Following stimulation with IL-1beta and TNFalpha, pro-inflammatory mediators as well as IGF-binding proteins were down-regulated more effectively in the presence of both genes to levels comparable to the non-stimulated control. Further, cartilage regeneration proteins type II collagen and proteoglycans were up-regulated in stimulated cells transfected with IGF-1 alone and in combination w...

Optimization of shRNA inhibitors by variation of the terminal loop sequence.

Antiviral Research — Wed, 24 Feb 2010 00:00:00 +0100

Authors: Schopman NC, Liu YP, Konstantinova P, Ter Brake O, Berkhout B Gene silencing by RNA interference (RNAi) can be achieved by intracellular expression of a short hairpin RNA (shRNA) that is processed into the effective small interfering RNA (siRNA) inhibitor by the RNAi machinery. Previous studies indicate that shRNA molecules do not always reflect the activity of corresponding synthetic siRNAs that attack the same target sequence. One obvious difference between these two effector molecules is the hairpin loop of the shRNA. Most studies use the original shRNA design of the pSuper system, but no extensive study regarding optimization of the shRNA loop sequence has been performed. We tested the impact of different hairpin loop sequences, varying in size and structure, on the activi...

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Adeno-Associated Virus-Mediated Rhodopsin Replacement Provides Therapeutic Benefit in Mice with a Targeted Disruption of the Rhodopsin Gene

Human Gene Therapy — Tue, 23 Feb 2010 04:06:14 +0100

Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)

Cell and gene therapy strategies for the treatment of postmyocardial infarction ventricular arrhythmias

Annals of the New York Academy of Sciences — Tue, 23 Feb 2010 00:00:00 +0100

Ventricular arrhythmias in the setting of a healed myocardial infarction represent a major cause of morbidity and mortality. The underlying mechanism is the presence of slow conduction tissue within the infarct border zone. In the current review we describe experimental gene and cell therapy approaches targeting the electrophysiologic substrate of the border zone, with the aim of preventing postinfarction ventricular arrhythmias. These include strategies that aim to prevent reentry by improving conduction velocity or by prolonging refractoriness. Attempts to augment conduction velocity include cardiomyocyte transplantation to regenerate the infarct, overexpression of unique sodium channels (to improve excitability), and methods to improve cell-to-cell coupling. Strategies to prolong refrac...

High Transgene Expression by Lentiviral Vectors Causes Maldevelopment of Purkinje Cells In Vivo.

Cerebellum — Tue, 23 Feb 2010 00:00:00 +0100

Authors: Sawada Y, Kajiwara G, Iizuka A, Takayama K, Shuvaev AN, Koyama C, Hirai H Lentiviral vectors are promising as gene-transfer vehicles for gene therapy targeted to intractable brain diseases. Although lentiviral vectors are thought to exert little toxicity on infected cells, the adverse influence of viral infection on vulnerable developing neurons has not been well studied. Here, we examined whether lentiviral vector infection and subsequent transgene expression affected the morphological and functional maturation of vigorously developing cerebellar Purkinje cells in vivo. Lentiviral vectors expressing GFP under the control of the murine stem cell virus (MSCV) promoter were injected into the cerebellar cortex of neonatal rat pups. Three weeks after treatment, GFP-expressing Purk...

Gene delivery using dimethyldidodecylammonium bromide-coated PLGA nanoparticles.

Biomaterials — Tue, 23 Feb 2010 00:00:00 +0100

Authors: Fay F, Quinn DJ, Gilmore BF, McCarron PA, Scott CJ In this present work we describe a poly(lactic-co-glycolic acid) (PLGA) nanoparticle formulation for intracellular delivery of plasmid DNA. This formulation was developed to encapsulate DNA within PLGA nanoparticles that combined salting out and emulsion-evaporation processes. This process reduced the requirement for sonication which can induce degradation of the DNA. A monodispersed nanoparticle population with a mean diameter of approximately 240 nm was produced, entrapping a model plasmid DNA in both supercoiled and open circular structures. To induce endosomal escape of the nanoparticles, a superficial cationic charge was introduced using positively charged surfactants cetyl trimethylammonium bromide (CTAB) and dimethyldid...

Preparation of pharmaceutical-grade plasmid DNA using methacrylate monolithic columns.

Vaccine — Tue, 23 Feb 2010 00:00:00 +0100

Authors: Smrekar F, Podgornik A, Ciringer M, Kontrec S, Raspor P, Strancar A, Peterka M Plasmid DNA (pDNA) used in vaccination and gene therapy has to be highly pure and homogenous, which point out necessity to develop efficient, reproducible and scalable downstream process. Convective Interaction Media (CIM) monolithic chromatographic supports being designed for purification of large molecules and nanoparticles seem to be a matrix of choice for pDNA purification. In present work we describe a pDNA purification process designed on two different CIM monolithic columns, based on anion-exchange (AEX) chromatography and hydrophobic interaction chromatography (HIC) chemistry. HIC monolith enabled separation of supercoiled (sc) pDNA from open circular (oc) pDNA, genomic DNA (gDNA) and endoto...

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Flightless Mosquitoes May Curb Dengue

MedlinePlus Health News — Mon, 22 Feb 2010 22:30:32 +0100

Genetically altered mosquitoes that cannot fly may help slow the spread of dengue fever and could be a harmless alternative to chemical insecticides, U.S. and British scientists said on Monday. Source: Reuters Health Related MedlinePlus Topics: Dengue, Genes and Gene Therapy, International Health (Source: MedlinePlus Health News)

Genetic Mutation Linked to Prostate Cancer in Blacks

MedlinePlus Health News — Mon, 22 Feb 2010 21:00:00 +0100

Finding may explain higher incidence among blacks than whites Source: HealthDay Related MedlinePlus Topics: African-American Health, Genes and Gene Therapy, Prostate Cancer (Source: MedlinePlus Health News)

The Role of Vascular Endothelial Growth Factor Gene as the Genetic Marker of Atherothrombotic Disorders and in the Gene Therapy of Coronary Artery Disease

Mon, 22 Feb 2010 13:57:56 +0100

(Source: Cardiovascular & Hematological Agents in Medicinal Chemistry (Formerly Current Medicinal Chemistry - Cardiovascular & Hematological Agents))

Gene Therapy Shows Promise Against HIV

MedicineNet HIV General — Mon, 22 Feb 2010 07:00:00 +0100

Title: Gene Therapy Shows Promise Against HIVCategory: Health NewsCreated: 2/19/2010 4:10:00 PMLast Editorial Review: 2/22/2010 (Source: MedicineNet HIV General)

Mobilization of Hematopoietic Stem Cells in a Thalassemic Mouse Model: Implications for Human Gene Therapy of Thalassemia

Human Gene Therapy — Sat, 20 Feb 2010 04:08:24 +0100

Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)

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Modification of Integration Site Preferences of an HIV-1-Based Vector by Expression of a Novel Synthetic Protein

Human Gene Therapy — Fri, 19 Feb 2010 22:20:47 +0100

Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)

Gene Therapy Shows Promise Against HIV

MedlinePlus Health News — Fri, 19 Feb 2010 22:00:00 +0100

Early findings suggest it could work, but application remains years away Source: HealthDay Related MedlinePlus Topics: AIDS, Genes and Gene Therapy (Source: MedlinePlus Health News)

Phase II HIV gene therapy trial has encouraging results

ScienceDaily Headlines — Fri, 19 Feb 2010 13:00:00 +0100

In a new phase II study using gene therapy to combat HIV, seven of eight subjects experienced a decrease in viral load set point and one subject experienced prolonged, complete control of HIV viremia for more than 14 weeks in the absence of HAART. The study looked at Lexgenleucel-T infusions in HIV-1 infected individuals prior to being taken off their antiretroviral treatment regimens as part of the study design's scheduled treatment interruption. (Source: ScienceDaily Headlines)

Phase II HIV Gene Therapy Trial Data At CROI 2010 Presented By Penn Researchers

Health News from Medical News Today — Fri, 19 Feb 2010 13:00:00 +0100

Researchers from the University of Pennsylvania School of Medicine presented the results from an ongoing Phase I/II open-label clinical trial of Lexgenleucel-T at the 16th Conference on Retroviruses and Opportunistic Infections (CROI) in San Francisco, CA. Lexgenleucel-T is a cell and gene therapy product being investigated for the treatment of HIV infection. The current study examined the effect of Lexgenleucel-T infusions in HIV-1 infected individuals prior to being taken off their antiretroviral treatment (HAART) regimens as part of the study design's scheduled treatment interruption... (Source: Health News from Medical News Today)

Phase II HIV Gene Therapy Trial Data At CROI 2010 Presented By Penn Researchers

HIV / AIDS News From Medical News Today — Fri, 19 Feb 2010 13:00:00 +0100

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Morphologic and functional changes in the unilateral 6-hydroxydopamine lesion rat model for Parkinson’s disease discerned with μSPECT and quantitative MRI

Magnetic Resonance Materials in Physics, Biology and Medicine — Fri, 19 Feb 2010 06:43:05 +0100

Conclusions These PD-related asymmetry ratios might be the result of counteracting changes in both intact and affected striatum and allowed us to diagnose PD lesions. As lateralization is known to occur also in PD patients and might be expected in transgenic PD models as well, we propose that MR-derived asymmetry ratios in the striatum might be a useful tool for in vivo phenotyping of animal models of PD. Content Type Journal ArticleCategory Research ArticleDOI 10.1007/s10334-010-0198-7Authors Nadja Van Camp, University of Antwerp Bio-Imaging Lab Groenenborgerlaan 171 2020 Antwerp BelgiumRuth Vreys, University of Antwerp Bio-Imaging Lab Groenenborgerlaan 171 2020 Antwerp BelgiumKoen Van Laere, University Hospital Leuven and K.U. Leuven Division of Nuclear Medicine ...

Long-Distance Runners May Have Endurance in Their Genes

MedlinePlus Health News — Thu, 18 Feb 2010 22:00:00 +0100

'Some of us are truly born to run,' researcher says Source: HealthDay Related MedlinePlus Topics: Genes and Gene Therapy, Sports Fitness (Source: MedlinePlus Health News)

Scientists Spot Genetic 'Fingerprints' of Individual Cancers

MedlinePlus Health News — Thu, 18 Feb 2010 22:00:00 +0100

Discovery could help doctors track course of disease, treatment response Source: HealthDay Related MedlinePlus Topics: Cancer, Genes and Gene Therapy, Genetic Testing (Source: MedlinePlus Health News)

Gene Therapy in Parkinsons Disease: Rationale and Current Status

CNS Drugs — Thu, 18 Feb 2010 02:06:21 +0100

(Source: CNS Drugs)

Characterization of a molecular switch system that regulates gene expression in mammalian cells through a small molecule

BMC Biotechnology - Latest articles — Thu, 18 Feb 2010 00:00:00 +0100

Conclusions: We have successfully shown that this system can induce tightly controlled transgene expression and can be used for transient transfections or retroviral transductions in mammalian cell culture. Further characterization is needed for gene therapy applications. (Source: BMC Biotechnology - Latest articles)

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Characterization of transgene expression in adenoviral-vector based HIV-1 vaccine candidates

Virology Journal — Thu, 18 Feb 2010 00:00:00 +0100

Recombinant adenovirus vectors have been extensively used in gene therapy clinical studies. More recently, the capability of inducing potent cell-mediated and humoral immunity has made these vectors equally attractive candidates for prophylactic or therapeutic vaccine applications. Merck developed HIV-1 vaccine candidates based on adenovirus serotype 5 (Ad5) vectors in which the E1 gene, a critical component for adenovirus replication, was replaced by the cytomegalovirus immediate/early promoter, followed by mutated versions of the HIV-1 gag, pol or nef genes (constructs referred to as MRKAd5gag, MRKAd5pol and MRKAd5nef, respectively). Vaccine performance was evaluated in vitro in a novel assay that measures the level of transgene expression in non-permissive A549 cells. Various combinatio...

Immunohistochemical detection of transgene expression in the brain using small epitope tags

BMC Biotechnology - Latest articles — Thu, 18 Feb 2010 00:00:00 +0100

Conclusions: We show here that several small epitope tags are useful for immunohistochemical detection of exogenous proteins in vivo. Our study also provides a generic methodology which is broadly applicable for the detection of overexpressed transgenes in mammalian brain tissue. (Source: BMC Biotechnology - Latest articles)

Scientists Map Genetic Regulatory Elements for the Heart

MedlinePlus Health News — Wed, 17 Feb 2010 22:48:58 +0100

Source: National Center for Biotechnology Information, National Heart, Lung, and Blood Institute, National Human Genome Research Institute Related MedlinePlus Topic: Genes and Gene Therapy (Source: MedlinePlus Health News)

Genome Study Shows What Cancers Have in Common

MedlinePlus Health News — Wed, 17 Feb 2010 21:08:48 +0100

Genetic abnormalities -- missing DNA or duplicate DNA -- that fuel the growth of one type of cancer may actually be at work in several others, U.S. researchers said on Wednesday. Source: Reuters Health Related MedlinePlus Topics: Cancer, Genes and Gene Therapy (Source: MedlinePlus Health News)