MedWorm: Gene Therapy

Gene Therapy Proves Effective In Treating Blindness

Health News from Medical News Today — Wed, 08 Feb 2012 23:00:00 +0100

Researchers from the Perelman School of Medicine at the University of Pennsylvania and The Children's Hospital of Philadelphia have conducted a recent study, published in Science Translational Medicine which focuses on gene therapy for congenital blindness. The scientists were able to improve sight in 3 adult patients who had previously been treated in one eye. The researchers used the same treatment on the second eye of the patients, and they were able to see in low-light situations and also find their way around. There were no conflicting effects reported... (Source: Health News from Medical News Today)

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More Success With Gene Therapy for Blindness

The Doctors Lounge - Health News — Wed, 08 Feb 2012 22:00:00 +0100

Repeat treatment improves vision for three adults with rare eye disease (Source: The Doctors Lounge - Health News)

Gene therapy 'gave me sight back'

BBC News | Health | UK Edition — Wed, 08 Feb 2012 19:35:29 +0100

Three US citizens who lost their sight in childhood have reported a dramatic improvement in vision after having gene therapy in both eyes. (Source: BBC News | Health | UK Edition)

Gene Therapy Improves Vision in Second Eye of 3 AdultsGene Therapy Improves Vision in Second Eye of 3 Adults

Medscape Today Headlines — Wed, 08 Feb 2012 19:27:34 +0100

The RPE65 gene delivered in an adeno-associated virus vector has safely improved vision in a second eye for patients with Leber congenital amaurosis type 2. Medscape Medical News (Source: Medscape Today Headlines)

Gene Therapy Helps People With Inherited Blindness See

WebMD Health — Wed, 08 Feb 2012 19:20:03 +0100

Functionally blind for many years, Tami Morehouse calls the gene therapy that partially restored her sight nothing short of a miracle. (Source: WebMD Health)

Targeting HLA class I expression to increase tumor immunogenicity

Tissue Antigens — Wed, 08 Feb 2012 11:12:45 +0100

The dynamic interaction between the host immune system and growing cancer has been of central interest to the field of tumor immunology over the past years. Recognition of tumor‐associated antigens (TAA) by self‐HLA (human leukocyte antigen) class I‐restricted CD8+ T cells is a main feature in the detection and destruction of malignant cells. The discovery and molecular characterization of TAA has changed the field of cancer treatment and introduced a new era of cancer immunotherapy aimed at increasing tumor immunogenicity and T‐cell‐mediated anti‐tumor immunity. Unfortunately, while these new protocols of cancer immunotherapy are mediating induction of tumor‐specific T lymphocytes in patients with certain malignancies, they have not yet delivered substantial clinical benefit...

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BioResearch Open Access launching in March 2012 from Mary Ann Liebert, Inc., publishers

EurekAlert! - Medicine and Health — Wed, 08 Feb 2012 05:00:00 +0100

(Mary Ann Liebert, Inc./Genetic Engineering News) BioResearch Open Access, a new bimonthly peer-reviewed open access journal, will launch in March 2012 by Mary Ann Liebert, Inc., publishers. The journal will provide a new rapid-publication forum for a broad range of scientific topics including but not limited to molecular and cellular biology, tissue engineering and biomaterials, regenerative medicine, stem cells, gene therapy, systems biology, genetics, biochemistry, virology, microbiology, and neuroscience. (Source: EurekAlert! - Medicine and Health)

Gene Research Offers Clues to Parkinson's Disease

MedlinePlus Health News — Tue, 07 Feb 2012 17:00:00 +0100

Key mutation in certain DNA is tied to a minority of cases of the illness, study says Source: HealthDay Related MedlinePlus Pages: Genes and Gene Therapy, Parkinson's Disease (Source: MedlinePlus Health News)

Gene Regulator in Brain's Executive Hub Tracked across Lifespan

MedlinePlus Health News — Mon, 06 Feb 2012 22:39:33 +0100

Source: National Institute of Mental Health - Related MedlinePlus Page: Genes and Gene Therapy (Source: MedlinePlus Health News)

Vestibular Regeneration – Experimental Models and Clinical Implications

Journal of Cellular and Molecular Medicine — Fri, 03 Feb 2012 05:00:00 +0100

AbstractTherapies aimed at the protection and/or regeneration of inner ear hair cells are of great interest given the significant monetary and quality of life impact of balance disorders. Different viral vectors have been shown to transfect various cell types in the inner ear. The past decade has provided tremendous advances in the use of adenoviral vectors to achieve targeted treatment delivery. Several routes of delivery have been identified in order to introduce vectors into the inner ear while minimizing injury to surrounding structures. Recently, the transcription factor Atoh1 was determined to play a critical role in hair cell differentiation. Adenoviral‐mediated overexpression of Atoh1 in culture and in vivo has demonstrated the ability to regenerate vestibular hair cells by causi...

Complementary treatment of siTERT for improving the antitumor effect of TERT-specific I-131 therapy

Cancer Gene Therapy — Fri, 03 Feb 2012 05:00:00 +0100

Authors: S Kim, H Youn, M G Song, J H Kang, H K Chung, D S Lee & J-K Chung (Source: Cancer Gene Therapy)

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Same Genes Key to Early & Late-Onset Alzheimer's

MedlinePlus Health News — Thu, 02 Feb 2012 14:00:00 +0100

Individual risk factors may influence when the brain disorder develops, researchers say Source: HealthDay Related MedlinePlus Pages: Alzheimer's Disease, Genes and Gene Therapy (Source: MedlinePlus Health News)

A novel lentiviral vector targets gene transfer into human hematopoietic stem cells in marrow from patients with bone marrow failure syndrome and in vivo in humanized mice

Blood — Thu, 02 Feb 2012 05:00:00 +0100

In conclusion, this new LV will facilitate HSC-based gene therapy by directly targeting these primitive cells in BM aspirates or total cord blood. Most importantly, in the future, RDTR/SCF-LVs might completely obviate ex vivo handling and simplify gene therapy for many hematopoietic defects because of their applicability to direct in vivo inoculation. (Source: Blood)

Cardiac gene therapy in large animals: bridge from bench to bedside

Gene Therapy — Thu, 02 Feb 2012 05:00:00 +0100

Authors: K Ishikawa, L Tilemann, D Ladage, J Aguero, L Leonardson, K Fish & Y Kawase (Source: Gene Therapy)

Nucleofection induces transient eIF2α phosphorylation by GCN2 and PERK

Gene Therapy — Thu, 02 Feb 2012 05:00:00 +0100

Nucleofection induces transient eIF2α phosphorylation by GCN2 and PERK Gene Therapy advance online publication, February 2, 2012. doi:10.1038/gt.2012.5 Authors: B R Anderson, K Karikó & D Weissman (Source: Gene Therapy)

Transcription of promoter from the human APRIL gene regulated by Sp1 and NF-kB.

Neoplasma — Thu, 02 Feb 2012 05:00:00 +0100

In this report, the promoter region of the APRIL gene was determined and the major transcription factor was investigated for the first time. Deletion analysis of 5'-ﬂanking region of the human APRIL gene and transient transfection revealed that a 538 bp region (from -1539 to -1001) was essential for promoter activation of the APRIL gene. The data from electrophoretic mobility shift assays (EMSA) indicated that the 538 bp promoter region was responsive to the specificity protein 1 (Sp1) and nuclear factor kappa B (NF-kB). Overexpression of Sp1 or NF-kB increased the activity of the APRIL promoter. Mithramycin A (inhibitor of Sp1) and Bay11-7082 (inhibitor of NF-kB) exhibited an inhibitory activity to APRIL promoter. Our results will benefit to the APRIL gene regulation investigation...

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RNA interference-mediated silencing of Stat5 induces apoptosis and growth suppression of hepatocellular carcinoma cells.

Neoplasma — Thu, 02 Feb 2012 05:00:00 +0100

In this study, we used human hepatocellular carcinoma cell line SMMC7721 as a model to demonstrate that Stat5 was highly expressed in these cells. Next we showed that RNAi mediated Stat5 knockdown could inhibit the proliferation and induce the apoptosis of SMMC7721 cells in vitro. Furthermore, we demonstrated that Stat5 knockdown inhibited the growth and induced the apoptosis of SMMC7721 cells in xenografts in nude mice. Taken together, our in vitro and in vivo data suggest that Stat5 plays an important role in human hepatocellular carcinoma. Inhibition of Stat5 by RNAi holds promise to be a novel gene therapy vector for hepatocellular carcinoma. Keywords: hepatocellular carcinoma; SMMC7721; RNA interference; Stat5; apoptosis. PMID: 22296499 [PubMed - as supplied by publisher] (Sou...

Adenovirus-mediated delivery of CALR and MAGE-A3 inhibits invasion and angiogenesis of glioblastoma cell line U87

Journal of Experimental and Clinical Cancer Research — Wed, 01 Feb 2012 05:00:00 +0100

Conclusion: Although Ad-CALR/MAGE-A3 and Ad-CALR demonstrated antiangiogenic effects on U87 cells, the repression of invasion was significant only in Ad-CALR/MAGE-A3-treated cells. To our knowledge, this is the first description of a role for combined CALR and MAGE-A3 in the anti-invasion and antiangiogenesis of U87. (Source: Journal of Experimental and Clinical Cancer Research)

Synergistic effects of CNS‐directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis

Annals of Neurology — Wed, 01 Feb 2012 05:00:00 +0100

Gene therapy: An eye for gene repair

Nature — Wed, 01 Feb 2012 05:00:00 +0100

Nature 482, 7383 (2012). doi:10.1038/482008e Gene therapy in dogs can reverse retinal defects that lead to blindness in humans.William Beltran and Gustavo Aguirre at the University of Pennsylvania in Philadelphia and their group targeted a form of retinitis pigmentosa. This is a common cause of blindness in which mutations (Source: Nature)

Single-step capture and sequencing of natural DNA for detection of BRCA1 mutations [METHOD]

Genome Research — Wed, 01 Feb 2012 05:00:00 +0100

Genetic testing for disease risk is an increasingly important component of medical care. However, testing can be expensive, which can lead to patients and physicians having limited access to the genetic information needed for medical decisions. To simplify DNA sample preparation and lower costs, we have developed a system in which any gene can be captured and sequenced directly from human genomic DNA without amplification, using no proteins or enzymes prior to sequencing. Extracted whole-genome DNA is acoustically sheared and loaded in a flow cell channel for single-molecule sequencing. Gene isolation, amplification, or ligation is not necessary. Accurate and low-cost detection of DNA sequence variants is demonstrated for the BRCA1 gene. Disease-causing mutations as well as common variants...

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Basic fibroblast growth factor modulates cell cycle of human umbilical cord‐derived mesenchymal stem cells

Cell Proliferation — Wed, 01 Feb 2012 05:00:00 +0100

ConclusionOur study showed that bFGF supplementation of UC‐MSC culture enhanced the cells' growth kinetics without compromising their nature. (Source: Cell Proliferation)

Science Shows How Exercise Might Help in Prostate Cancer

MedlinePlus Health News — Wed, 01 Feb 2012 00:00:00 +0100

Activity-induced genetic changes could slow or prevent disease progression, researchers say Source: HealthDay Related MedlinePlus Pages: Exercise and Physical Fitness, Genes and Gene Therapy, Prostate Cancer (Source: MedlinePlus Health News)

Syndecan-4 proteoliposomes enhance fibroblast growth factor-2 (FGF-2)-induced proliferation, migration, and neovascularization of ischemic muscle [Medical Sciences]

Proceedings of the National Academy of Sciences — Tue, 31 Jan 2012 05:00:00 +0100

Ischemia of the myocardium and lower limbs is a common consequence of arterial disease and a major source of morbidity and mortality in modernized countries. Inducing neovascularization for the treatment of ischemia is an appealing therapeutic strategy for patients for whom traditional treatment modalities cannot be performed or are ineffective. In the past, the stimulation of blood vessel growth was pursued using direct delivery of growth factors, angiogenic gene therapy, or cellular therapy. Although therapeutic angiogenesis holds great promise for treating patients with ischemia, current methods have not found success in clinical trials. Fibroblast growth factor-2 (FGF-2) was one of the first growth factors to be tested for use in therapeutic angiogenesis. Here, we present a method for ...

Gene Study Sheds Light on Body Clock's Link to Diabetes

MedlinePlus Health News — Mon, 30 Jan 2012 19:00:00 +0100

A mutation affecting sleep-wake hormone may disrupt blood sugar control, researchers find Source: HealthDay Related MedlinePlus Pages: Diabetes Type 2, Genes and Gene Therapy, Hormones (Source: MedlinePlus Health News)

Downregulation of apoptosis-inducing factor in Harlequin mice induces progressive and severe optic atrophy which is durably prevented by AAV2-AIF1 gene therapy

Brain — Sat, 28 Jan 2012 05:00:00 +0100

The Harlequin mutant mouse, characterized by loss of function of apoptosis-inducing factor, represents a reliable genetic model that resembles pathologies caused by human mitochondrial complex I deficiency. Therefore, we extensively characterized the retinal morphology and function of Harlequin mice during the course of neuronal cell death leading to blindness, with the aim of preventing optic atrophy. Retinas and optic nerves from these mice showed an isolated respiratory chain complex I defect correlated with retinal ganglion cell loss, optic atrophy, glial and microglial cell activation. All of these changes led to irreversible vision loss. In control mice, retinas AIF1 messenger RNA was 2.3-fold more abundant than AIF2, both messenger RNAs being sorted to the mitochondrial surface. In ...

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Efficient and rapid uptake of magnetic carbon nanotubes into human monocytic cells: implications for cell-based cancer gene therapy.

Biotechnology Letters — Sat, 28 Jan 2012 05:00:00 +0100

Authors: Gul-Uludag H, Lu W, Xu P, Xing J, Chen J Abstract Monocyte-based gene therapies in cancer have been hampered by either the resistance of these cells to non-viral molecular delivery methods or their poor trafficking to the tumor site after their ex vivo manipulations. Magnetic nanoparticles (MNP)-loaded genetically engineered monocytes can efficiently delivered to tumor site by external magnetic field, but they are not ideal delivery tools due to their spherical shape. Hence, we have investigated the cellular uptake efficiency and cytotoxicity of fluorescein isothiocyanate (FITC)-labelled magnetic carbon nanotubes (FITC-mCNT) in human monocytic leukemia cell line THP-1 for application in cell-based gene therapy against cancer. Uptake of FITC-mCNT into THP-1 cells reached 10...

Semireplication-competent vesicular stomatitis virus as a novel platform for oncolytic virotherapy

Journal of Molecular Medicine — Fri, 27 Jan 2012 17:53:02 +0100

In conclusion, srVSV is a promising platform for virotherapeutic approaches and also for VSV-based vector vaccines, combining improved safety with an increased coding capacity for therapeutic transgenes, potentially allowing for multipronged approaches. Content Type Journal ArticleCategory Original ArticlePages 1-12DOI 10.1007/s00109-012-0863-6Authors Alexander Muik, Georg-Speyer-Haus, 60596 Frankfurt am Main, GermanyCatherine Dold, Institute for Virology, Innsbruck Medical University, Fritz-Pregl-Str. 3, A-6020 Innsbruck, AustriaYvonne Geiß, Georg-Speyer-Haus, 60596 Frankfurt am Main, GermanyAndreas Volk, Georg-Speyer-Haus, 60596 Frankfurt am Main, GermanyMarina Werbizki, Georg-Speyer-Haus, 60596 Frankfurt am Main, GermanyUrsula Dietrich, Georg-Speyer-Haus, 60596 Frankfurt am Mai...

Hypoxia imaging predicts success of hypoxia-induced cytosine deaminase/5-fluorocytosine gene therapy in a murine lung tumor model

Cancer Gene Therapy — Fri, 27 Jan 2012 05:00:00 +0100

Hypoxia imaging predicts success of hypoxia-induced cytosine deaminase/5-fluorocytosine gene therapy in a murine lung tumor model Cancer Gene Therapy advance online publication, January 27, 2012. doi:10.1038/cgt.2011.87 Authors: B-F Lee, C-H Lee, N-T Chiu, C-C Hsia, L-H Shen & A-L Shiau (Source: Cancer Gene Therapy)

Adenovirus-mediated Aurora A shRNA driven by stathmin promoter suppressed tumor growth and enhanced paclitaxel chemotherapy sensitivity in human breast carcinoma cells

Cancer Gene Therapy — Fri, 27 Jan 2012 05:00:00 +0100

Authors: M Long, G Yin, L Liu, F Lin, X Wang, J Ren, J Wei, K Dong & H Zhang (Source: Cancer Gene Therapy)

Antitumor efficacy of oncolytic herpes simplex virus adsorbed onto antigen-specific lymphocytes

Cancer Gene Therapy — Fri, 27 Jan 2012 05:00:00 +0100

Authors: A Kanzaki, H Kasuya, K Yamamura, T T Sahin, N Nomura, T Shikano, T Shirota, G Tan, S Fukuda, M Misawa, Y Nishikawa, S Yamada, T Fujii, H Sugimoto, S Nomoto, S Takeda, Y Kodera & A Nakao (Source: Cancer Gene Therapy)

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The Effects of Anti-Inflammatory and Anti-Angiogenic DNA Vaccination on Diabetic Nephropathy in Rats

Human Gene Therapy — Fri, 27 Jan 2012 04:09:18 +0100

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

Researchers Spot Potential Bile Duct Cancer Drug Targets

MedlinePlus Health News — Thu, 26 Jan 2012 22:00:00 +0100

Mutations in two genes found in tumors appear to fuel their growth Source: HealthDay Related MedlinePlus Pages: Bile Duct Cancer, Genes and Gene Therapy (Source: MedlinePlus Health News)

Factors Linked to Age of Onset of Menopause Identified

MedlinePlus Health News — Thu, 26 Jan 2012 21:00:00 +0100

Researchers zero in on 13 new genetic clues Source: HealthDay Related MedlinePlus Pages: Genes and Gene Therapy, Menopause (Source: MedlinePlus Health News)

Mutations in 2 Genes Linked to Rare Autism-Related Disorder

MedlinePlus Health News — Thu, 26 Jan 2012 19:00:00 +0100

Research sheds light on Joubert syndrome, which causes physical and mental disabilities Source: HealthDay Related MedlinePlus Pages: Cerebellar Disorders, Genes and Gene Therapy (Source: MedlinePlus Health News)

Improving TCR Gene Therapy for Treatment of Haematological Malignancies

Advances in Pharmacological Sciences — Thu, 26 Jan 2012 18:56:20 +0100

Adoptive immunotherapy using TCR gene modified T cells may allow separation of beneficial Graft versus tumour responses from harmful GvHD. Improvements to this include methods to generate high avidity or high affinity TCR, improvements in vector design and reduction in mispairing. Following adoptive transfer, TCR transduced T cells must be able to survive and persist in vivo to give most effective antitumour responses. Central memory or naive T cells have both been shown to be more effective than effector cells at expanding and persisting in vivo. Lymphodepletion may enhance persistence of transferred T cell populations. TCR gene transfer can be used to redirect CD4 helper T cells, and these could be used in combination with CD8+ tumour specific T cells to provide help for the antitumour r...

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Differentiation of GDNF and NT-3 dual gene-modified rat bone marrow mesenchymal stem cells into enteric neuron-like cells

Journal of Huazhong University of Science and Technology -- Medical Sciences -- — Thu, 26 Jan 2012 16:49:56 +0100

This study provides an experimental basis for gene therapy to treat enteric nervous system-related disorders, such as HD. Content Type Journal ArticlePages 87-91DOI 10.1007/s11596-012-0015-9Authors Heyun Gao, Department of Pediatric Surgery, Huazhong University of Science and Technology, Wuhan, 430030 ChinaMingfa Wei, Department of Pediatric Surgery, Huazhong University of Science and Technology, Wuhan, 430030 ChinaYan Wang, Department of Otorhinolaryngology Head and Neck Surgery, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430030 ChinaXiaojuan Wu, Department of Pediatric Surgery, Huazhong University of Science and Technology, Wuhan, 430030 ChinaTianqi Zhu, Department of Pediatric Surgery, Huazhong University of Science and Technolo...

Successful Gene Therapy in Utero for Lethal Murine Hypophosphatasia

Human Gene Therapy — Thu, 26 Jan 2012 16:45:42 +0100

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

Gene Therapy for Retinitis Pigmentosa Caused by MFRP Mutations: Human Phenotype and Preliminary Proof of Concept

Human Gene Therapy — Thu, 26 Jan 2012 16:45:38 +0100

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

Long-Term Expression of Human Coagulation Factor VIII in a Tolerant Mouse Model Using the φC31 Integrase System

Human Gene Therapy — Thu, 26 Jan 2012 16:45:34 +0100

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

Decreasing Disease Severity in Symptomatic, Smn−/−;SMN2+/+, Spinal Muscular Atrophy Mice Following scAAV9-SMN Delivery

Human Gene Therapy — Thu, 26 Jan 2012 16:40:10 +0100

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

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Characterization of an HIV-Targeted Transcriptional Gene-Silencing RNA in Primary Cells

Human Gene Therapy — Thu, 26 Jan 2012 16:40:06 +0100

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

Prednisolone Treatment Does Not Interfere with 2′-O-Methyl Phosphorothioate Antisense-Mediated Exon Skipping in Duchenne Muscular Dystrophy

Human Gene Therapy — Thu, 26 Jan 2012 16:37:12 +0100

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

Safety and Tolerability of Magnetic Resonance Imaging-Guided Convection-Enhanced Delivery of AAV2-hAADC with a Novel Delivery Platform in Nonhuman Primate Striatum

Human Gene Therapy — Thu, 26 Jan 2012 16:35:16 +0100

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

Enhanced Athletic Performance on Multisite AAV-IGF1 Gene Transfer Coincides with Massive Modification of the Muscle Proteome

Human Gene Therapy — Thu, 26 Jan 2012 16:35:12 +0100

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

Syngeneic Hematopoietic Stem Cell Transplantation Enhances the Antitumor Immunity of Intratumoral Type I Interferon Gene Transfer for Sarcoma

Human Gene Therapy — Thu, 26 Jan 2012 16:31:59 +0100

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

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A 'Disruptive Science' Ready For Commercial Development - Gene Therapy

Health News from Medical News Today — Thu, 26 Jan 2012 08:00:00 +0100

The time for commercial development of gene therapy has come. Patients with diseases treatable and curable with gene therapy deserve access to the technology, which has demonstrated both its effectiveness and feasibility, says James Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy in a provocative commentary and accompanying videocast. Human Gene Therapy and Human Gene Therapy (HGT) Methods are peer-reviewed journals published by Mary Ann Liebert, Inc.. Until recently, gene therapy has been reserved for severe diseases with few treatment options... (Source: Health News from Medical News Today)

AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage

Blood — Thu, 26 Jan 2012 05:00:00 +0100

We explored adeno-associated viral vector (AAV)–mediated gene transfer in the perinatal period in animal models of severe congenital factor VII (FVII) deficiency, a disease associated with early postnatal life-threatening hemorrhage. In young adult mice with plasma FVII < 1% of normal, a single tail vein administration of AAV (1 x 1013 vector genomes [vg]/kg) resulted in expression of murine FVII at 266% ± 34% of normal for ≥ 67 days, which mediated protection against fatal hemorrhage and significantly improved survival. Codon optimization of human FVII (hFVIIcoop) improved AAV transgene expression by 37-fold compared with the wild-type hFVII cDNA. In adult macaques, a single peripheral vein injection of 2 x 1011 vg/kg of the hFVIIcoop AAV vector resulted in therapeutic ...

Reprogramming axonal behavior by axon-specific viral transduction

Gene Therapy — Thu, 26 Jan 2012 05:00:00 +0100

Authors: B A Walker, U Hengst, H J Kim, N L Jeon, E F Schmidt, N Heintz, T A Milner & S R Jaffrey (Source: Gene Therapy)

AAV-based shRNA silencing of NF-κB ameliorates muscle pathologies in mdx mice

Gene Therapy — Thu, 26 Jan 2012 05:00:00 +0100

AAV-based shRNA silencing of NF-κB ameliorates muscle pathologies in mdx mice Gene Therapy advance online publication, January 26, 2012. doi:10.1038/gt.2011.207 Authors: Q Yang, Y Tang, K Imbrogno, A Lu, J D Proto, A Chen, F Guo, F H Fu, J Huard & B Wang (Source: Gene Therapy)

Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems

Gene Therapy — Thu, 26 Jan 2012 05:00:00 +0100

Authors: C N Mattar, S N Waddington, A Biswas, N Johana, X W Ng, A S Fisk, N M Fisk, L G Tan, A A Rahim, S M K Buckley, M H Tan, J Lu, M Choolani & J K Y Chan (Source: Gene Therapy)

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Can gene therapy be used to prevent vein graft disease?

University of Bristol news — Wed, 25 Jan 2012 10:45:29 +0100

Gavin Murphy, Reader in Cardiac Surgery in the School of Clinical Sciences at the University of Bristol, has been awarded a grant of over £100,000 from Heart Research UK to investigate gene therapy in vein grafts that are used in heart bypass surgery. (Source: University of Bristol news)

Researchers Develop Gene Therapy That Could Correct A Common Form Of Blindness

Health News from Medical News Today — Wed, 25 Jan 2012 09:00:00 +0100

A new gene therapy method developed by University of Florida researchers has the potential to treat a common form of blindness that strikes both youngsters and adults. The technique works by replacing a malfunctioning gene in the eye with a normal working copy that supplies a protein necessary for light-sensitive cells in the eye to function. The findings are published in the Proceedings of the National Academy of Sciences online. Several complex and costly steps remain before the gene therapy technique can be used in humans, but once at that stage, it has great potential to change lives... (Source: Health News from Medical News Today)

French institute prepares for gene-therapy push

Nature — Wed, 25 Jan 2012 05:00:00 +0100

Nature 481, 7382 (2012). http://www.nature.com/doifinder/10.1038/481423a Author: Alison Abbott Genethon relaunches itself as a force for translational medicine. (Source: Nature)

Label-free quantitative analysis for studying the interactions between nanoparticles and plasma proteins.

Analytical and Bioanalytical Chemistry — Wed, 25 Jan 2012 05:00:00 +0100

Authors: Capriotti AL, Caracciolo G, Caruso G, Cavaliere C, Pozzi D, Samperi R, Laganà A Abstract A shotgun proteomics approach was used to compare human plasma protein binding capability with cationic liposomes, DNA-cationic lipid complexes (lipoplexes), and lipid-polycation-DNA (LPD) complexes. Nano-high-performance liquid chromatography coupled with a high-resolution LTQ Orbitrap XL mass spectrometer was used to characterize and compare their protein corona. Spectral counting and area under curve methods were used to perform label-free quantification. Substantial qualitative and quantitative differences were found among proteins bound to the three different systems investigated. Protein variety found on lipoplexes and LPD complexes was richer than that found on cationic liposom...

Gene Mutations May Boost Ovarian Cancer Survival

MedlinePlus Health News — Tue, 24 Jan 2012 21:00:00 +0100

Women with BRCA1, BRCA2 have higher risk for the disease, but prognosis might be better Source: HealthDay Related MedlinePlus Pages: Genes and Gene Therapy, Ovarian Cancer (Source: MedlinePlus Health News)

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Retinitis Pigmentosa In Dogs Cured By Gene Therapy

Health News from Medical News Today — Tue, 24 Jan 2012 10:00:00 +0100

Members of a University of Pennsylvania research team have shown that they can prevent, or even reverse, a blinding retinal disease, X-linked Retinitis Pigmentosa, or XLRP, in dogs. The disease in humans and dogs is caused by defects in the RPGR gene and results in early, severe and progressive vision loss. It is one of the most common inherited forms of retinal degeneration in man... (Source: Health News from Medical News Today)

Stem Cells Show Promise For Delivering Gene Therapy For Huntington's Disease

Health News from Medical News Today — Tue, 24 Jan 2012 08:00:00 +0100

A team of researchers at the UC Davis Institute for Regenerative Cures has developed a technique for using stem cells to deliver therapy that specifically targets the genetic abnormality found in Huntington's disease, a hereditary brain disorder that causes progressive uncontrolled movements, dementia and death. The findings, now available online in the journal Molecular and Cellular Neuroscience, suggest a promising approach that might block the disease from advancing... (Source: Health News from Medical News Today)

Gene Transfer of c-met Confers Protection Against d-Galactosamine/Lipopolysaccharide-Induced Acute Liver Failure

Digestive Diseases and Sciences — Tue, 24 Jan 2012 06:48:45 +0100

Conclusions This study provides the initial evidence that c-met may serve as a novel target for gene therapy, and may be of clinical benefit in the treatment of patients with ALF. Content Type Journal ArticleCategory Original ArticlePages 1-10DOI 10.1007/s10620-012-2052-4Authors Chuanlong Zhu, Department of Infectious Diseases, Affiliated Provincial Hospital, Anhui Medical University, Hefei, 230001 ChinaYuwen Li, Department of Pediatrics, Affiliated Provincial Hospital, Anhui Medical University, Hefei, 230001 ChinaWenting Li, Department of Infectious Diseases, Affiliated Provincial Hospital, Anhui Medical University, Hefei, 230001 ChinaQuan Wu, Department of Central Lab, Affiliated Provincial Hospital, Anhui Medical University, Hefei, 230001 ChinaRentao Gao, Departmen...

Gene therapy is a 'disruptive science' ready for commercial development

EurekAlert! - Medicine and Health — Tue, 24 Jan 2012 05:00:00 +0100

(Mary Ann Liebert, Inc./Genetic Engineering News) The time for commercial development of gene therapy has come. Patients with diseases treatable and curable with gene therapy deserve access to the technology, which has demonstrated both its effectiveness and feasibility, says James Wilson, M.D., Ph.D., Editor-in-Chief of Human Gene Therapy in a provocative commentary and accompanying videocast. (Source: EurekAlert! - Medicine and Health)

UK sets sights on gene therapy in eggs

Nature — Tue, 24 Jan 2012 05:00:00 +0100

Nature 481, 7382 (2012). http://www.nature.com/doifinder/10.1038/481419a Author: Ewen Callaway Public consultation and safety assessment would pave the way for embryo manipulation to treat genetic diseases. (Source: Nature)

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Animal models for studying vein graft failure and therapeutic interventions.

Current Opinion in Pharmacology — Tue, 24 Jan 2012 05:00:00 +0100

Authors: Thomas AC Abstract Vein grafts have been extensively used to bypass blockages in arteries, but are themselves subject to early closure by thrombosis or later obstruction by vein graft disease (neointimal hyperplasia and remodelling). Animal models are a crucial means of testing potential therapeutic and surgical interventions to prevent graft stenosis and occlusion. This review outlines many of the animal models of vein grafting. Recent studies include targeted gene therapy to prevent acute vein graft thrombosis and the use of folic acid to limit graft failure in diabetic pigs. PMID: 22281067 [PubMed - as supplied by publisher] (Source: Current Opinion in Pharmacology)

An arrayed human genomic library constructed in the PAC shuttle vector pJCPAC-Mam2 for genome-wide association studies and gene therapy.

Gene — Tue, 24 Jan 2012 05:00:00 +0100

Authors: Fuesler J, Nagahama Y, Szulewski J, Mundorff J, Bireley S, Coren JS Abstract The various iterations of the HapMap Project and many genome-wide association studies (GWAS) have identified hundreds of potential genes involved in monogenic and multifactorial traits. We constructed an arrayed 115,000-member human genomic library in the PAC shuttle vector pJCPAC-Mam2 that can be propagated in both bacterial and human cells. The library appears to represent a two-fold coverage of the human genome. Transient transfection of a p53-containing PAC clone into p53-null Saos-2 human osteosarcoma cells demonstrated that both p53 mRNA and protein were produced. Additionally, expression of the p53 protein triggered apoptosis in a subset of the Saos-2 cells. This library should serve as a v...

Researchers develop gene therapy that could correct a common form of blindness

ScienceDaily Headlines — Mon, 23 Jan 2012 21:34:34 +0100

A new gene therapy has the potential to treat a common form of blindness that strikes both youngsters and adults. The technique works by replacing a malfunctioning gene in the eye with a normal working copy that supplies a protein necessary for light-sensitive cells in the eye to function. Several complex steps remain before the gene therapy technique can be used in humans, but once at that stage, it has great potential to change lives. (Source: ScienceDaily Headlines)

Gene therapy cures retinitis pigmentosa in dogs

ScienceDaily Headlines — Mon, 23 Jan 2012 20:25:25 +0100

Scientists have shown that they can prevent, or even reverse, a blinding retinal disease, X-linked Retinitis Pigmentosa, or XLRP, in dogs. The disease in humans and dogs is caused by defects in the RPGR gene and results in early, severe and progressive vision loss. It is one of the most common inherited forms of retinal degeneration in man. (Source: ScienceDaily Headlines)

Researchers develop gene therapy that could correct a common form of blindness

EurekAlert! - Medicine and Health — Mon, 23 Jan 2012 05:00:00 +0100

(University of Florida) A new gene therapy has the potential to treat a common form of blindness that strikes both youngsters and adults. The technique works by replacing a malfunctioning gene in the eye with a normal working copy that supplies a protein necessary for light-sensitive cells in the eye to function. Several complex steps remain before the gene therapy technique can be used in humans, but once at that stage, it has great potential to change lives. (Source: EurekAlert! - Medicine and Health)

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New Genetic Clues to Breast Cancer?

MedlinePlus Health News — Sun, 22 Jan 2012 19:00:00 +0100

Researchers say they've identified three more genomic regions associated with the diseaseSource: HealthDay Related MedlinePlus Pages: Breast Cancer, Genes and Gene Therapy (Source: MedlinePlus Health News)

Efficient growth suppression and apoptosis in human laryngeal carcinoma cell line HEP‐2 induced by an adeno‐associated virus expressing human FAS ligand

Head and Neck — Fri, 20 Jan 2012 05:00:00 +0100

ConclusionOur findings suggest that the introduction of FasL into head and neck squamous cell carcinoma may induce significant apoptosis, and adeno‐associated virus may be a useful vehicle for gene therapy. © 2012 Wiley Periodicals, Inc. Head Neck, 2012 (Source: Head and Neck)

Sendai virus vector‐mediated brain‐derived neurotrophic factor expression ameliorates memory deficits and synaptic degeneration in a transgenic mouse model of Alzheimer's disease

Journal of Neuroscience Research — Thu, 19 Jan 2012 12:23:37 +0100

AbstractGrowing evidence suggests that decreased brain‐derived neurotrophic factor (BDNF) levels are associated with Alzheimer's disease (AD) pathogenesis. Therefore, BDNF gene therapy is considered to be a promising therapeutic strategy for treating AD. Sendai virus (SeV) is a type I parainfluenza virus that does not interact with host chromosomes because of its strict cytoplasmic life cycle. Although SeV is nonpathogenic in primates, including humans, its infectivity for neurons is strong. Here we demonstrate that SeV vectors effectively infected neurons, even though they were injected into subcortical white matter. Moreover, SeV vectors significantly induced BDNF expression, ameliorating synaptic degeneration and memory deficits in a transgenic mouse model of AD (Tg2576). This is the ...

CD27 costimulation augments the survival and antitumor activity of redirected human T cells in vivo

Blood — Thu, 19 Jan 2012 05:00:00 +0100

The costimulatory effects of CD27 on T lymphocyte effector function and memory formation has been confined to evaluations in mouse models, in vitro human cell culture systems, and clinical observations. Here, we tested whether CD27 costimulation actively enhances human T-cell function, expansion, and survival in vitro and in vivo. Human T cells transduced to express an antigen-specific chimeric antigen receptor (CAR-T) containing an intracellular CD3 zeta (CD3) chain signaling module with the CD27 costimulatory motif in tandem exerted increased antigen-stimulated effector functions in vitro, including cytokine secretion and cytotoxicity, compared with CAR-T with CD3 alone. After antigen stimulation in vitro, CD27-bearing CAR-T cells also proliferated, up-regulated Bcl-XL protein expression...

Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs

Gene Therapy — Thu, 19 Jan 2012 05:00:00 +0100

Authors: B L Ellis, M L Hirsch, S N Porter, R J Samulski & M H Porteus (Source: Gene Therapy)

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BRCA1 gene therapy reduces systemic inflammatory response and multiple organ failure and improves survival in experimental sepsis

Gene Therapy — Thu, 19 Jan 2012 05:00:00 +0100

Authors: H Teoh, A Quan, A K Creighton, K W Annie Bang, K K Singh, P C Shukla, N Gupta, Y Pan, F Lovren, H Leong-Poi, M Al-Omran & S Verma (Source: Gene Therapy)

Minicircle DNA electrotransfer for efficient tissue-targeted gene delivery

Gene Therapy — Thu, 19 Jan 2012 05:00:00 +0100

Authors: S Chabot, J Orio, M Schmeer, M Schleef, M Golzio & J Teissié (Source: Gene Therapy)

Rapid titration of retroviral vectors using a β-lactamase protein fragment complementation assay

Gene Therapy — Thu, 19 Jan 2012 05:00:00 +0100

Rapid titration of retroviral vectors using a β-lactamase protein fragment complementation assay Gene Therapy advance online publication, January 19, 2012. doi:10.1038/gt.2011.212 Authors: W Ou, M P Marino, C Lu & J Reiser (Source: Gene Therapy)

The Sodium Iodide Symporter (NIS) as an Imaging Reporter for Gene, Viral, and Cell-based Therapies.

Current Gene Therapy — Thu, 19 Jan 2012 05:00:00 +0100

Authors: Penheiter AR, Russell SJ, Carlson SK Abstract Preclinical and clinical tomographic imaging systems increasingly are being utilized for non-invasive imaging of reporter gene products to reveal the distribution of molecular therapeutics within living subjects. Reporter gene and probe combinations can be employed to monitor vectors for gene, viral, and cell-based therapies. There are several reporter systems available; however, those employing radionuclides for positron emission tomography (PET) or single-photon emission computed tomography (SPECT) offer the highest sensitivity and the greatest promise for deep tissue imaging in humans. Within the category of radionuclide reporters, the thyroidal sodium iodide symporter (NIS) has emerged as one of the most promising for precl...

PET Imaging For Gene & Cell Therapy.

Current Gene Therapy — Thu, 19 Jan 2012 05:00:00 +0100

Authors: Collins SA, Hiraoka K, Inagaki A, Kasahara N, Tangney M Abstract As the interest in gene therapy increases, the development of an efficient and reliable means to monitor gene delivery and expression in patients is becoming more important. An ideal imaging modality would be non-invasive, allowing for repeated imaging, thus validating stages subsequent to vector administration and allowing for the improvement of clinical protocols. Positron Emission Tomography (PET) has been employed for some time in clinical imaging and has in more recent years been adapted to enable imaging in small animal models, including gene therapy models for a range of diseases. PET imaging is based on the detection of trace quantities of positron-emitting molecular probe within cells post-administra...

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The bacterial lux reporter system: applications in bacterial localisation studies.

Current Gene Therapy — Thu, 19 Jan 2012 05:00:00 +0100

Authors: Gahan CG Abstract Bacterial production of visible light is a natural phenomenon occurring in marine (Vibrio and Photobacterium) and terrestrial (Photorhabdus) species. The mechanism underpinning light production in these organisms is similar and involves the oxidation of an aldehyde substrate in a reaction catalysed by the bacterial luciferase enzyme. The genes encoding the luciferase and a fatty acid reductase complex which synthesizes the substrate are contained in a single operon (the lux operon). This provides a useful reporter system as cloning the operon into a recipient host bacterium will generate visible light without the requirement to add exogenous substrate. The light can be detected in vivo in the living animal using a sensitive detection system and is therefo...

In vivo Optical Imaging in Gene & Cell Therapy.

Current Gene Therapy — Thu, 19 Jan 2012 05:00:00 +0100

Authors: Tangney M, Francis KP Abstract Integral to the development of all gene therapy technologies is the ability to monitor gene delivery, in terms of distribution, levels and kinetics of vector transgene expression. This can be achieved to some extent at the preclinical level through use of traditional ex vivo analytical methods, but these hold several drawbacks, not least the requirement for death of experimental subjects for such end-point assays. Real-time In vivo analysis of reporter gene expression empowers the investigator with the ability to non-invasively assess gene delivery over time, as well as host responses to vector administration and therapeutic interventions. While there exist several technologies for such small animal monitoring, imaging of light emission from ...

Infusing CD19-Directed T Cells to Augment Disease Control in Patients Undergoing Autologous Hematopoietic Stem-Cell Transplantation for Advanced B-Lymphoid Malignancies

Human Gene Therapy — Thu, 19 Jan 2012 04:07:40 +0100

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

Study Maps Path from Smoking to Emphysema in Mice

MedlinePlus Health News — Wed, 18 Jan 2012 21:00:00 +0100

Immune response to smoke causes the disease, researchers say Source: HealthDay Related MedlinePlus Pages: Genes and Gene Therapy, Smoking (Source: MedlinePlus Health News)

Genes May Guide Intelligence Throughout Life

MedlinePlus Health News — Wed, 18 Jan 2012 19:00:00 +0100

Some people stay smart as they age in part because of genetic factors, study suggests Source: HealthDay Related MedlinePlus Page: Genes and Gene Therapy (Source: MedlinePlus Health News)

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Genes May Give Clues to Severe form of Lupus

MedlinePlus Health News — Wed, 18 Jan 2012 17:00:00 +0100

A DNA sequence known as an 'accelerator' might explain some aggressive cases, researchers say Source: HealthDay Related MedlinePlus Pages: Genes and Gene Therapy, Lupus (Source: MedlinePlus Health News)

Pediatric thyroid cancer

Seminars in Pediatric Surgery — Wed, 18 Jan 2012 06:36:29 +0100

Thyroid cancer is an uncommon childhood malignancy that presents primarily in young children or adolescent females and may be related to radiation exposure or genetic predisposition. Gene alterations, such as RET mutation or RET/PTC rearrangement, are not uncommon. Recent studies have lead to an increased understanding of the role of these particular gene alterations in the diagnosis, prognosis, and treatment of thyroid cancer. Surgery remains the mainstay of treatment for thyroid cancer followed by radioactive iodine when appropriate. In patients with MEN2, prophylactic thyroidectomy is recommended, although a delay in the initial diagnosis is common. With early aggressive treatment and long-term follow-up, these patients generally have excellent outcomes. Recent research suggests potenti...

Infusing CD19-Directed T Cells to Augment Disease Control in Patients Undergoing Autologous Hematopoietic Stem-Cell Transplantation for Advanced B-Lymphoid Malignancies

Human Gene Therapy — Wed, 18 Jan 2012 04:07:35 +0100

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

Your Taste for Fat May Reside in Your Genes

MedlinePlus Health News — Mon, 16 Jan 2012 17:00:00 +0100

Individual sensitivity might influence what and how much you eat, small study suggests Source: HealthDay Related MedlinePlus Pages: Dietary Fats, Genes and Gene Therapy (Source: MedlinePlus Health News)

Clinical Progress in Gene Therapy: Sustained Partial Correction of the Bleeding Disorder in Patients Suffering from Severe Hemophilia B

Human Gene Therapy — Mon, 16 Jan 2012 16:53:11 +0100

Human Gene Therapy Jan 2012, Vol. 23, No. 1: 4-6. (Source: Human Gene Therapy)

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Inactivation of Transcription Factor Pit-1 to Target Tumoral Somatolactotroph Cells

Human Gene Therapy — Mon, 16 Jan 2012 16:53:06 +0100

Human Gene Therapy Jan 2012, Vol. 23, No. 1: 104-114. (Source: Human Gene Therapy)

A Potent Oncolytic Adenovirus Selectively Blocks the STAT3 Signaling Pathway and Potentiates Cisplatin Antitumor Activity in Ovarian Cancer

Human Gene Therapy — Mon, 16 Jan 2012 16:53:02 +0100

Human Gene Therapy Jan 2012, Vol. 23, No. 1: 32-45. (Source: Human Gene Therapy)

Native Molecular State of Adeno-Associated Viral Vectors Revealed by Single-Molecule Sequencing

Human Gene Therapy — Mon, 16 Jan 2012 16:52:58 +0100

Human Gene Therapy Jan 2012, Vol. 23, No. 1: 46-55. (Source: Human Gene Therapy)

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The European Hospital Exemption Clause—New Option for Gene Therapy?

Human Gene Therapy — Mon, 16 Jan 2012 16:52:55 +0100

Human Gene Therapy Jan 2012, Vol. 23, No. 1: 7-12. (Source: Human Gene Therapy)

Response to Intra-Arterial Oncolytic Virotherapy with the Herpes Virus NV1020 Evaluated by [18F]Fluorodeoxyglucose Positron Emission Tomography and Computed Tomography

Human Gene Therapy — Mon, 16 Jan 2012 16:52:53 +0100

Human Gene Therapy Jan 2012, Vol. 23, No. 1: 91-97. (Source: Human Gene Therapy)

The New Self-Inactivating Lentiviral Vector for Thalassemia Gene Therapy Combining Two HPFH Activating Elements Corrects Human Thalassemic Hematopoietic Stem Cells

Human Gene Therapy — Mon, 16 Jan 2012 16:52:51 +0100

Human Gene Therapy Jan 2012, Vol. 23, No. 1: 15-31. (Source: Human Gene Therapy)

A Transductionally Retargeted Adenoviral Vector for Virotherapy of Her2/neu-Expressing Prostate Cancer

Human Gene Therapy — Mon, 16 Jan 2012 16:52:42 +0100

Human Gene Therapy Jan 2012, Vol. 23, No. 1: 70-82. (Source: Human Gene Therapy)

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It's Time for Gene Therapy to Get Disruptive!

Human Gene Therapy — Mon, 16 Jan 2012 16:52:38 +0100

Human Gene Therapy Jan 2012, Vol. 23, No. 1: 1-3. (Source: Human Gene Therapy)

Gene Therapy Briefs

Human Gene Therapy — Mon, 16 Jan 2012 16:52:38 +0100

Human Gene Therapy Jan 2012, Vol. 23, No. 1: 13-14. (Source: Human Gene Therapy)

It's Time for Gene Therapy to Get Disruptive!

Human Gene Therapy — Mon, 16 Jan 2012 16:52:38 +0100

Human Gene Therapy Jan 2012, Vol. 23, No. 1: 1-3. (Source: Human Gene Therapy)

Gene Therapy Briefs

Human Gene Therapy — Mon, 16 Jan 2012 16:52:38 +0100

Human Gene Therapy Jan 2012, Vol. 23, No. 1: 13-14. (Source: Human Gene Therapy)

Distribution Properties of Lentiviral Vectors Administered into the Striatum by Convection-Enhanced Delivery

Human Gene Therapy — Mon, 16 Jan 2012 16:52:35 +0100

Human Gene Therapy Jan 2012, Vol. 23, No. 1: 115-127. (Source: Human Gene Therapy)

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Human Involucrin Promoter Mediates Repression-Resistant and Compartment-Specific LEKTI Expression

Human Gene Therapy — Mon, 16 Jan 2012 16:52:29 +0100

Human Gene Therapy Jan 2012, Vol. 23, No. 1: 83-90. (Source: Human Gene Therapy)

Hyaluronidase and Collagenase Increase the Transfection Efficiency of Gene Electrotransfer in Various Murine Tumors

Human Gene Therapy — Mon, 16 Jan 2012 16:52:26 +0100

Human Gene Therapy Jan 2012, Vol. 23, No. 1: 128-137. (Source: Human Gene Therapy)

Efficient Generation of A9 Midbrain Dopaminergic Neurons by Lentiviral Delivery of LMX1A in Human Embryonic Stem Cells and Induced Pluripotent Stem Cells

Human Gene Therapy — Mon, 16 Jan 2012 16:52:22 +0100

Human Gene Therapy Jan 2012, Vol. 23, No. 1: 56-69. (Source: Human Gene Therapy)

Novel Mini–Dystrophin Gene Dual Adeno-Associated Virus Vectors Restore Neuronal Nitric Oxide Synthase Expression at the Sarcolemma

Human Gene Therapy — Mon, 16 Jan 2012 16:52:11 +0100

Human Gene Therapy Jan 2012, Vol. 23, No. 1: 98-103. (Source: Human Gene Therapy)

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Negative regulation-resistant p53 variant enhances oncolytic adenoviral gene therapy.

Herpes — Mon, 16 Jan 2012 05:00:00 +0100

Authors: Koo T, Choi IK, Kim M, Lee JS, Oh E, Kim J, Yun CO Abstract Intact p53 function is essential for responsiveness to cancer therapy. However, p53 activity is attenuated by the proto-oncoprotein Mdm2, the adenovirus protein E1B-55kD, and the p53 C-terminal domain. To confer resistance to Mdm2, E1B-55kD, and C-terminal negative regulation, we generated a p53 variant (p53VPΔ30) by deleting the N-terminal and C-terminal regions of wild-type (wt) p53 and inserting the transcriptional activation domain of herpes simplex virus VP16 protein. The oncolytic adenovirus vector Ad-mΔ19 expressing p53VPΔ30 (Ad-m19/p53VPΔ30) showed greater cytotoxicity than Ad-mΔ19 expressing wt p53 or other p53 variants in human cancer cell lines. We found that Ad-m∆19/p53VPΔ30 induced apopt...

Smooth Muscle Cell Polymeric Transfection is an Efficient Alternative to Traditional Methods of Experimental Gene Therapy

Journal of Surgical Research — Sat, 14 Jan 2012 22:30:18 +0100

(Source: Journal of Surgical Research)

Cell- and gene-based approaches to tendon regeneration

Journal of Shoulder and Elbow Surgery — Sat, 14 Jan 2012 14:39:44 +0100

Repair of rotator cuff tears in experimental models has been significantly improved by the use of enhanced biologic approaches, including platelet-rich plasma, bone marrow aspirate, growth factor supplements, and cell- and gene-modified cell therapy. Despite added complexity, cell-based therapies form an important part of enhanced repair, and combinations of carrier vehicles, growth factors, and implanted cells provide the best opportunity for robust repair. Bone marrow–derived mesenchymal stem cells provide a stimulus for repair in flexor tendons, but application in rotator cuff repair has not shown universally positive results. The use of scaffolds such as platelet-rich plasma, fibrin, and synthetic vehicles and the use of gene priming for stem cell differentiation and local anabolic a...

Cytokines in rotator cuff degeneration and repair

Journal of Shoulder and Elbow Surgery — Sat, 14 Jan 2012 14:39:40 +0100

The pathogenesis of rotator cuff degeneration remains poorly defined, and the incidence of degenerative tears is increasing in the aging population. Rates of recurrent tear and incomplete tendon-to-bone healing after repair remain significant for large and massive tears. Previous studies have documented a disorganized, fibrous junction at the tendon-to-bone interface after rotator cuff healing that does not recapitulate the organization of the native enthesis. Many biologic factors have been implicated in coordinating tendon-to-bone healing and maintenance of the enthesis after rotator cuff repair, including the expression and activation of transforming growth factor-β, basic fibroblast growth factor, platelet-derived growth factor-β, matrix metalloproteinases, and tissue inhibitors of m...

Biologic approaches to enhance rotator cuff healing after injury

Journal of Shoulder and Elbow Surgery — Sat, 14 Jan 2012 14:39:37 +0100

Conclusions: Use of growth factors, stem cell therapy, and other tissue-engineering means serve to augment classical surgical rotator cuff repair procedures. The combination of stem cells and growth factors resulted in enhanced repair that emulated uninjured tissue, but the literature search reflected paucity of research in this field. Preclinical evidence from gene therapy and stem cell studies can be used as a start to move therapy from the experimental phase to clinical translation in patients.Level of evidence: Review Article. (Source: Journal of Shoulder and Elbow Surgery)

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Senescence (ageing) @ 2011

Indian Journal of Dermatology — Sat, 14 Jan 2012 05:00:00 +0100

Anjana NigamIndian Journal of Dermatology 2011 56(6):615-621Ageing, also called as senescence, is one of the most complex, intrinsic, biological processes of growing older and resulting into reduced functional ability of the organism. Telomerase, environment, low calorie diets, free radicals, etc., are all believed to affect this ageing process. A number of genetic components of ageing have been identified using model organisms. Genes, mainly the sirtuins, regulate the ageing speed by indirection and controlling organism resistance to damages by exogenous and endogenous stresses. In higher organisms, ageing is likely to be regulated, in part, through the insulin/insulin-like growth factor 1 pathway. Besides this, the induction of apoptosis in stem and progenitor cells, increased p53 activi...

Do plasma proteins distinguish between liposomes of varying charge density?

Journal of Proteomics — Sat, 14 Jan 2012 05:00:00 +0100

Authors: Capriotti AL, Caracciolo G, Cavaliere C, Foglia P, Pozzi D, Samperi R, Laganà A Abstract Cationic liposomes (CLs) are one of the most employed nonviral nanovector systems in gene therapy. However, their transfection efficiency is strongly affected by interactions with plasma components, that lead to the formation of a "protein corona" onto CL surface. The interactions between nanoparticles entering the body and biomolecules have an essential role for their biodistribution. Because the knowledge of proteins adsorbed onto vector surface could be useful in the screening of new, more efficient and more biocompatible liposomal formulations, the behavior of three CLs with different membrane charge densities was investigated. The proteins of the three coronas were identified by ...

MicroRNA-34a affects the occurrence of laryngeal squamous cell carcinoma by targeting the antiapoptotic gene survivin

Medical Oncology — Fri, 13 Jan 2012 16:51:25 +0100

Abstract Survivin has been shown to be an ideal target for cancer gene therapy because of its strong antiapoptotic effect. MicroRNA-34a (miR-34a) can function as a tumor suppressor in some cancers through negative regulation of gene expression. However, the relationship between miR-34a and survivin in larynx squamous cell carcinoma (LSCC) has not been explored. The abundance of survivin mRNA and miR-34a in LSCC tissues were measured using quantitative real-time polymerase chain reaction. Their expression levels were analyzed and correlated with tumor differentiation, lymphatic metastasis, clinical stages, and survival rates. MiR-34a mimic was transfected using liposomes to increase its level in LSCC cancer cell line, Hep-2. The effects of miR-34a on survivin protein expres...

Gene therapy.

Herpes — Fri, 13 Jan 2012 11:30:16 +0100

Authors: Gomez-Manzano C, Jiang H, Alonso M, Yung WK, Fueyo J Abstract During the last 10 years, gene therapy for brain tumors has known peaks and valleys. The first attempts to induce therapeutic effect, using retrovirus to transduce the HSV-TK gene and adenovirus to transfer wild-type p53 cDNA, failed significantly to improve the survival of the patients. In both cases, the failure was attributed to vector deficiencies, also termed the 'vector gap'. To address the problem of delivery, investigators have moved from replication-deficient vectors to replication-competent, tumor-selective viruses. These viruses are currently being tested in the clinical setting. In this review, we discuss the progress made with herpes simplex viruses (G207), reoviruses that naturally target Ras pathw...

Treatment with targeted vesicular stomatitis virus generates therapeutic multifunctional anti-tumor memory CD4 T cells

Cancer Gene Therapy — Fri, 13 Jan 2012 05:00:00 +0100

Authors: Y Gao, P Whitaker-Dowling, J A Griffin & I Bergman (Source: Cancer Gene Therapy)

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Regenerative Therapies for Improving Myocardial Perfusion in Patients with Cardiovascular Disease: Failure to Meet Expectations but Optimism for the Future.

Current Vascular Pharmacology — Fri, 13 Jan 2012 05:00:00 +0100

Authors: Sellke FW, Lassaletta AD, Robich MP, Chu LM, Ruel M Abstract Cardiovascular disease continues to be a major cause of death in the Western world and has been extending into areas previously seemingly immune to its effects. Catheter-based interventions and coronary artery bypass surgery have markedly improved cardiovascular health, but a number of patients with coronary artery disease cannot undergo repeated interventions or they receive an incomplete revascularization with standard revascularization methods, which has been associated with a poor clinical outcome. Despite early demonstration of improvement in myocardial perfusion and function with growth factor, gene therapy or cellular therapies, clinical studies have found little if any real benefit. The discordance betwee...

Pseudotype-Independent Nonspecific Uptake of Gammaretroviral and Lentiviral Particles in Human Cells

Human Gene Therapy — Fri, 13 Jan 2012 04:10:51 +0100

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

Improved Function of the Failing Rat Heart by Regulated Expression of Insulin-Like Growth Factor I via Intramuscular Gene Transfer

Human Gene Therapy — Thu, 12 Jan 2012 15:10:21 +0100

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

Pseudotype-Independent Nonspecific Uptake of Gammaretroviral and Lentiviral Particles in Human Cells

Human Gene Therapy — Thu, 12 Jan 2012 15:10:15 +0100

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

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Histone Deacetylase Inhibitors Restore Cell Surface Expression of the Coxsackie Adenovirus Receptor and Enhance CMV Promoter Activity in Castration-Resistant Prostate Cancer Cells

Computational Intelligence and Neuroscience — Thu, 12 Jan 2012 11:11:41 +0100

Adenoviral gene therapy using the death receptor ligand TRAIL as the therapeutic transgene can be safely administered via intraprostatic injection but has not been evaluated for efficacy in patients. Here we investigated the efficacy of adenoviral TRAIL gene therapy in a model of castration resistant prostate cancer and found that intratumoral injections can significantly delay tumor growth but cannot eliminate established lesions. We hypothesized that an underlying cause is inefficient adenoviral delivery. Using the LNCaP progression model of prostate cancer we show that surface CAR expression decreases with increasing tumorigenicity and that castration resistant C4-2b cells were more difficult to transduce with adenovirus than castration sensitive LNCaP cells. Many genes, including CAR, ...

Inflammation and Immune Response of Intra-Articular Serotype 2 Adeno-Associated Virus or Adenovirus Vectors in a Large Animal Model

Computational Intelligence and Neuroscience — Thu, 12 Jan 2012 11:11:41 +0100

Intra-articular gene therapy has potential for the treatment of osteoarthritis and rheumatoid arthritis. To quantify in vitro relative gene transduction, equine chondrocytes and synovial cells were treated with adenovirus vectors (Ad), serotype 2 adeno-associated virus vectors (rAAV2), or self-complementary (sc) AAV2 vectors carrying green fluorescent protein (GFP). Using 6 horses, bilateral metacarpophalangeal joints were injected with Ad, rAAV2, or scAAV2 vectors carrying GFP genes to assess the in vivo joint inflammation and neutralizing antibody (NAb) titer in serum and joint fluid. In vitro, the greater transduction efficiency and sustained gene expression were achieved by scAAV2 compared to rAAV2 in equine chondrocytes and synovial cells. In vivo, AAV2 demonstrated less joint inflamm...

Nucleic Acid-Based Therapy Approaches for Huntington's Disease

Computational Intelligence and Neuroscience — Thu, 12 Jan 2012 11:11:41 +0100

Huntington's disease (HD) is caused by a dominant mutation that results in an unstable expansion of a CAG repeat in the huntingtin gene leading to a toxic gain of function in huntingtin protein which causes massive neurodegeneration mainly in the striatum and clinical symptoms associated with the disease. Since the mutation has multiple effects in the cell and the precise mechanism of the disease remains to be elucidated, gene therapy approaches have been developed that intervene in different aspects of the condition. These approaches include increasing expression of growth factors, decreasing levels of mutant huntingtin, and restoring cell metabolism and transcriptional balance. The aim of this paper is to outline the nucleic acid-based therapeutic strategies that have been tested to ...

Tapping The Body's Own Defenses, Researchers Look To Cutting-Edge Gene Therapy For Bladder Cancer

Health News from Medical News Today — Thu, 12 Jan 2012 08:00:00 +0100

Bladder cancer, most frequently caused by smoking and exposure to carcinogens in the workplace, is one of the top 10 most common forms of cancer in men and women in the U.S. More than 70 percent of bladder cancers are diagnosed in stage T1 or less and have not invaded the muscle layer. At these early stages, standard treatment is surgery (transurethral resection) and the burning away of tumors with high energy electricity (fulguration). Many patients also may receive subsequent intravesical chemotherapy because there is often a high-risk for cancer recurrence... (Source: Health News from Medical News Today)

Aurora kinase A-specific T-cell receptor gene transfer redirects T lymphocytes to display effective antileukemia reactivity

Blood — Thu, 12 Jan 2012 05:00:00 +0100

In this study, we examined the feasibility of adoptive therapy using redirected T cells expressing an HLA-A*0201–restricted AURKA207-215-specific T-cell receptor (TCR). Retrovirally transduced T cells recognized relevant peptide-pulsed but not control target cells. Furthermore, TCR-redirected CD8+ T cells lysed AURKA-overexpressing human leukemic cells in an HLA-A*0201–restricted manner, but did not kill HLA-A*0201+ normal cells, including hematopoietic progenitors. In addition, AURKA207-215-specific TCR-transduced CD4+ T cells displayed target-responsive Th1 cytokine production. Finally, AURKA207-215-specific TCR-transduced CD8+ T cells displayed antileukemia efficacy in a xenograft mouse model. Collectively, these data demonstrate the feasibility of redirected T cell–ba...

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AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in mice

Gene Therapy — Thu, 12 Jan 2012 05:00:00 +0100

Authors: C Hu & G S Lipshutz (Source: Gene Therapy)

A simple detection system for adenovirus receptor expression using a telomerase-specific replication-competent adenovirus

Gene Therapy — Thu, 12 Jan 2012 05:00:00 +0100

Authors: T Sasaki, H Tazawa, J Hasei, S Osaki, T Kunisada, A Yoshida, Y Hashimoto, S Yano, R Yoshida, S Kagawa, F Uno, Y Urata, T Ozaki & T Fujiwara (Source: Gene Therapy)

Development of the Nanobody display technology to target lentiviral vectors to antigen-presenting cells

Gene Therapy — Thu, 12 Jan 2012 05:00:00 +0100

Authors: C Goyvaerts, K De Groeve, J Dingemans, S Van Lint, L Robays, C Heirman, J Reiser, X-Y Zhang, K Thielemans, P De Baetselier, G Raes & K Breckpot (Source: Gene Therapy)

LIM domain protein-3 (LMP3) cooperates with BMP7 to promote tissue regeneration by ligament progenitor cells

Gene Therapy — Thu, 12 Jan 2012 05:00:00 +0100

Authors: Z Lin, H F Rios, C-H Park, A D Taut, Q Jin, J V Sugai, P D Robbins & W V Giannobile (Source: Gene Therapy)

Engineered human Tmpk fused with truncated cell-surface markers: versatile cell-fate control safety cassettes

Gene Therapy — Thu, 12 Jan 2012 05:00:00 +0100

Authors: M Scaife, N Pacienza, B C Y Au, J C M Wang, S Devine, E Scheid, C-J Lee, O Lopez-Perez, A Neschadim, D H Fowler, R Foley & J A Medin (Source: Gene Therapy)

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A novel lentivirus for quantitative assessment of gene knockdown in stem cell differentiation

Gene Therapy — Thu, 12 Jan 2012 05:00:00 +0100

Authors: S Alimperti, P Lei, J Tian & S T Andreadis (Source: Gene Therapy)

Gene Research Sheds Light on Rare Immune Disease

MedlinePlus Health News — Wed, 11 Jan 2012 22:00:00 +0100

One symptom, cold urticaria, is a potentially serious disorder triggered by exposure to low temperatures Source: HealthDay Related MedlinePlus Pages: Genes and Gene Therapy, Immune System and Disorders (Source: MedlinePlus Health News)

Scientists Identify Inherited Prostate Cancer Gene

MedlinePlus Health News — Wed, 11 Jan 2012 22:00:00 +0100

Men with the mutation are at 10 to 20 times increased risk of prostate cancer Source: HealthDay Related MedlinePlus Pages: Genes and Gene Therapy, Prostate Cancer (Source: MedlinePlus Health News)

Humoral Immunity to AAV-6, 8, and 9 in Normal and Dystrophic Dogs

Human Gene Therapy — Wed, 11 Jan 2012 18:13:56 +0100

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

Oncolytic Measles Virus Encoding Thyroidal Sodium Iodide Symporter for Squamous Cell Cancer of the Head and Neck Radiovirotherapy

Human Gene Therapy — Wed, 11 Jan 2012 18:13:52 +0100

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

Survival In Brain Cancer Patients May Be Improved By Personalized Gene Therapies

Health News from Medical News Today — Wed, 11 Jan 2012 08:00:00 +0100

Personalized prognostic tools and gene-based therapies may improve the survival and quality of life of patients suffering from glioblastoma, an aggressive and deadly form of brain cancer, reports a new University of Illinois study funded by the NIH National Cancer Institute. "We confirmed known biomarkers of glioblastoma survival and discovered new general and clinical-dependent gene profiles," said Nicola Serao, a U of I Ph.D. candidate in animal sciences with a focus in statistical genomics... (Source: Health News from Medical News Today)

Emerging therapy for diabetic neuropathy: Cell therapy targeting vessels and nerves.

Endocrine, Metabolic and Immune Disorders Drug Targets — Wed, 11 Jan 2012 05:00:00 +0100

Authors: Kim H, Kim JJ, Yoon YS Abstract Diabetic neuropathy (DN), the most common complication of diabetes, frequently leads to foot ulcers and may progress to limb amputations. Despite continuous increase in incidences, there is no clinical therapy to effectively treat DN. Pathogenetically, DN is characterized by reduced vascularity in peripheral nerves and deficiency in angiogenic and neurotrophic factors. We will briefly review the pathogenic mechanisms of DN and the effects of cell therapies for DN. To reverse the changes of DN, studies have attempted neurotrophic or angiogenic factors for treatment in the form of protein or gene therapy; however, the effects were modest if not ineffective. Recent studies have demonstrated that bone marrow (BM)-derived cells such as mononuclea...

Lessons learned from 5 years of newborn screening for congenital adrenal hyperplasia in the Czech Republic: 17-hydroxyprogesterone, genotypes, and screening performance

European Journal of Pediatrics — Tue, 10 Jan 2012 16:59:58 +0100

Abstract The aims were to summarize the experience and to determine the performance metrics of newborn screening (NBS) for congenital adrenal hyperplasia (CAH) in the Czech Republic. 17-Hydroxyprogesterone (17OHP) was measured in NBS samples prospectively in 545,026 newborns and retrospectively in 31 CAH patients born outside the study period. A total of 2,811 screened newborns had abnormal 17OHP; CAH was confirmed in 46 probands. One patient with a severe–moderate genotype of CAH had 17OHP below the cut-off and was diagnosed clinically. This corresponds to a screening sensitivity of 98% and a false positive rate (FPR) of 0.51%. The median of 17OHP in the most severe genotypes was 484 nmol/L (n = 21); in severe/moderate, 321 nmol/L (n = 30); in moderate,...

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Tapping the body's own defenses, researchers look to cutting-edge gene therapy for bladder cancer

EurekAlert! - Medicine and Health — Tue, 10 Jan 2012 05:00:00 +0100

(University Hospitals Case Medical Center) The prognosis for recurrent cancer is poor, which drives clinician-scientists like William Larchian, M.D., orologic oncologist, University Hospitals Urology Institute at University Hospitals Case Medical Center, and associate professor of surgery, Case Western Reserve University School of Medicine, and his colleagues to develop an immunotherapy for bladder cancer that will stimulate the body's own natural defense mechanisms to cure the disease and prevent recurrence. (Source: EurekAlert! - Medicine and Health)

Progress in Gene and Cell Therapy for Cystic Fibrosis Lung Disease.

Current Pharmaceutical Design — Tue, 10 Jan 2012 05:00:00 +0100

Authors: Griesenbach U, Alton EW Abstract Although the development of gene therapy for cystic fibrosis (CF) was high priority for many groups in academia and industry in the first 10 to 15 years after cloning the gene, more recently active research into CF gene therapy is only being performed by a small number of committed, mainly academic, groups. However, despite the warning enthusiasm, which is largely due to the realisation that gene transfer into lungs is more difficult than originally thought and the fact that meaningful clinical trials are expensive and difficult to perform, gene therapy continues to hold promise for the treatment of CF lung disease. Problems related to repeat administration of adenovirus and adeno-associated virus-based vectors led to a focus on non-viral v...

Exercise May Help Those at Higher Risk for Alzheimer's

MedlinePlus Health News — Mon, 09 Jan 2012 21:00:00 +0100

Sedentary people with gene variation had more brain changes Source: HealthDay Related MedlinePlus Pages: Alzheimer's Disease, Exercise for Seniors, Genes and Gene Therapy (Source: MedlinePlus Health News)

Researchers Look at Genomes of Nonsmokers with Lung Cancer

MedlinePlus Health News — Mon, 09 Jan 2012 21:00:00 +0100

Never-smokers may have different mutations in genes than smokers, suggests small, early study Source: HealthDay Related MedlinePlus Pages: Genes and Gene Therapy, Lung Cancer (Source: MedlinePlus Health News)

Immune Reconstitution in Chronic Lymphocytic Leukemia

Current Hematologic Malignancy Reports — Mon, 09 Jan 2012 19:39:48 +0100

This article reviews the immune defect in CLL and discusses strategies aimed at repairing or circumventing this defect. In particular, it focuses on recent developments in the areas of CD40 ligand (CD40L/CD154) gene therapy, immunomodulatory agents such as lenalidomide, and adoptive transfer of T cells bearing chimeric antigen receptors. Content Type Journal ArticleCategory Chronic Lymphocytic Leukemia (S O'Brien, Section Editor)Pages 1-8DOI 10.1007/s11899-011-0106-xAuthors John C. Riches, Barts Cancer Institute, Queen Mary University of London, 3rd Floor John Vane Science Centre, Charterhouse Square, London, EC1M 6BQ UKAlan G. Ramsay, Barts Cancer Institute, Queen Mary University of London, 3rd Floor John Vane Science Centre, Charterhouse Square, London, EC1M 6BQ UKJohn G. Gribbe...

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Solving The Structure Of A Protein That Shows Promise As A DNA-targeting Molecule For Gene Therapy

Health News from Medical News Today — Mon, 09 Jan 2012 08:00:00 +0100

Researchers at Fred Hutchinson Cancer Research Center have solved the three-dimensional structure of a newly discovered type of gene-targeting protein that has shown to be useful as a DNA-targeting molecule for gene correction, gene therapy and gene modification. The findings are published online in Science Express on Jan. 5. Using a unique form of computational and X-ray crystallographic analyses, a team of researchers led by Barry L. Stoddard, Ph.D... (Source: Health News from Medical News Today)

Personalized gene therapies may increase survival in brain cancer patients

EurekAlert! - Medicine and Health — Mon, 09 Jan 2012 05:00:00 +0100

(University of Illinois College of Agricultural, Consumer and Environmental Sciences) Personalized prognostic tools and gene-based therapies may improve the survival and quality of life of patients suffering from glioblastoma, an aggressive and deadly form of brain cancer, reports a new University of Illinois study funded by the NIH National Cancer Institute. (Source: EurekAlert! - Medicine and Health)

Clinical gene therapies progress

Nature Biotechnology — Mon, 09 Jan 2012 05:00:00 +0100

Nature Biotechnology 30, 60 (2012). doi:10.1038/nbt.2102 Author: Laura DeFrancesco (Source: Nature Biotechnology)

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations: Safety and Efficacy in 15 Children and Adults Followed Up to 3 Years [Clinical Trial]

Archives of Opthalmology — Mon, 09 Jan 2012 05:00:00 +0100

Conclusions Gene therapy for Leber congenital amaurosis caused by RPE65 mutations is sufficiently safe and substantially efficacious in the extrafoveal retina. There is no benefit and some risk in treating the fovea. No evidence of age-dependent effects was found. Our results point to specific treatment strategies for subsequent phases. Application to Clinical Practice Gene therapy for inherited retinal disease has the potential to become a future part of clinical practice. Trial Registration clinicaltrials.gov Identifier: NCT00481546 (Source: Archives of Opthalmology)