Gene Therapy
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Perioperative Management of the Adult with Cystic Fibrosis.
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Since cystic fibrosis (CF) was first differentiated from celiac disease in 1938, the medical care of patients with CF has substantially improved. These improvements have resulted in a significant increase in median survival and the quality of life experienced by patients. The resultant increase in survival has caused the "average" CF patient to be a young adult and not a child. The gene that causes CF was first identified in 1989 and is the first gene discovered by positional cloning. Unfortunately, gene therapy for CF has not been successful, although it continues to hold great promise for future patient care. Althoug...
Source: Anesthesia and Analgesia - November 20, 2009 Category: Anesthesiology Authors: Huffmyer JL, Littlewood KE, Nemergut EC Tags: Anesth Analg Source Type: journals
Science: Michael Aminoff Discusses Gene Therapy for Parkinson's Disease
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Michael J. Aminoff, MD, DSc, FRCP, FAAN, talks about his recently published paper on safety and tolerability of putaminal AADC gene therapy for Parkinson's disease (Source: American Academy of Neurology)
Source: American Academy of Neurology - November 20, 2009 Category: Medical Law Source Type: organizations
Asthma Combo Seems Less Influenced By Genes
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Findings may not apply to blacks, researchers note Source: HealthDay
Related MedlinePlus Topics: Asthma, Genes and Gene Therapy (Source: MedlinePlus Health News)
Source: MedlinePlus Health News - November 20, 2009 Category: Consumer Health News Source Type: consumer
[Construction of an expression vector carrying short hairpin RNA targeting hTERT gene and its effects on breast cancer cell telomerase activity and proliferation in vivo.]
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CONCLUSION: Transfection with the recombinant plasmid containing the shRNA targeting hTERT gene can down-regulate telomerase activity and inhibit proliferation of breast cancer cells in vitro, suggesting the potential of gene therapy targeting telomerase in the treatment of breast cancer.
PMID: 19923062 [PubMed - as supplied by publisher] (Source: Journal of Southern Medical University)
Source: Journal of Southern Medical University - November 20, 2009 Category: Universities & Medical Training Authors: Liu XX, Yao C, Zhang H, Wang SM, Wang SM Tags: Nan Fang Yi Ke Da Xue Xue Bao Source Type: journals
Society for Hematology and Stem Cells Travel Grants to ISEH 2010 Meeting
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Date: 15 - 18 September, 2010Location: Melbourne Convention & Exhibition Centre, Melbourne Australia (www.mcec.com.au/)
Through the generous support of the State government of Victoria we are able to offer up to 200 travel grants for PhD students and post-doctoral delegates as follows:Delegates traveling from Europe AUD$2000Delegates traveling from the USA AUD$1500Delegates traveling from Asia AUD$1200Delegates traveling from New Zealand or within Australia AUD$300Applications need to be submitted with your abstract and will be assess...
Source: ScanGrants feed - November 19, 2009 Category: Research Authors: Society for Hematology and Stem Cells Source Type: funding
Moving Gene Therapy Forward with Mobile DNA
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Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)
Source: Human Gene Therapy - November 18, 2009 Category: Genetics & Stem Cells Tags: article Source Type: journals
Fragment C of tetanus toxin, more than a carrier. Novel perspectives in non-viral ALS gene therapy
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Abstract The non-toxic carboxy-terminal fragment of tetanus toxin heavy chain (TTC) has been implicated in the activation of cascades
responsible for trophic actions and neuroprotection by inhibition of apoptosis. Previous in vitro studies have described signalling
pathways that underlie the administration of TTC to neurons. We investigated whether these properties were maintained in a
mouse model of neurodegenerative disease. Naked DNA encoding for TTC was injected intramuscularly and neuromuscular function
and clinical behaviour were monitored until endstage in the transgenic SOD1G93A mouse model that expr...
Source: Journal of Molecular Medicine - November 18, 2009 Category: Molecular Biology Tags: Journal of Molecular Medicine Source Type: journals
Gene therapy with vascular endothelial growth factors.
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Therapeutic angiogenesis is a promising new concept for the treatment of myocardial and peripheral ischaemia. Members of the VEGF (vascular endothelial growth factor) family are among the most powerful modulators of angiogenesis. They regulate vascular growth and maintenance during embryogenesis and in adults. The present review summarizes the current status of therapeutic angiogenesis using VEGF, with special reference to preclinical studies.
PMID: 19909246 [PubMed - in process] (Source: Biochemical Society Transactions)
Source: Biochemical Society Transactions - November 18, 2009 Category: Biochemistry Authors: Ylä-Herttuala S Tags: Biochem Soc Trans Source Type: journals
Ligand-directed cancer gene therapy to angiogenic vasculature.
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Gene therapy strategies in cancer have remained an active area of preclinical and clinical research. One of the current limitations to successful trials is the relative transduction efficiency to produce a therapeutic effect. While intratumoral injections are the mainstay of many treatment regimens to date, this approach is hindered by hydrostatic pressures within the tumor and is not always applicable to all tumor subtypes. Vascular-targeting strategies introduce an alternative method to deliver vectors with higher local concentrations and minimization of systemic toxicity. Moreover, therapeutic targeting of angiogeni...
Source: Advances in Genetics - November 18, 2009 Category: Genetics & Stem Cells Authors: Driessen WH, Ozawa MG, Arap W, Pasqualini R Tags: Adv Genet Source Type: journals
Adeno-associated viral vectors and their redirection to cell-type specific receptors.
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Efficient and specific delivery of genes to the cell type of interest is a crucial issue in gene therapy. Adeno-associated virus (AAV) has gained particular interest as gene vector recently and is therefore the focus of this chapter. Its low frequency of random integration into the genome and the moderate immune response make AAV an attractive platform for vector design. Like in most other vector systems, the tropism of AAV vectors limits their utility for certain tissues especially upon systemic application. This may in part be circumvented by using AAV serotypes with an in vivo gene transduction pattern most closely ...
Source: Advances in Genetics - November 18, 2009 Category: Genetics & Stem Cells Authors: Michelfelder S, Trepel M Tags: Adv Genet Source Type: journals
Conditionally replicating adenovirus improves gene replication efficiency and anticancer effect of E1-deleted adenovirus carrying TRAIL in head and neck squamous cell carcinoma
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In conclusion, this combined strategy using CRAd and adenovirus carrying a therapeutic gene increased the gene transfer rate and enhanced antitumor effects. We expect that this combination strategy could be extended to a multitarget cancer gene therapy by combining multiple adenoviruses and CRAd. (Cancer Sci 2009) (Source: Cancer Science)
Source: Cancer Science - November 18, 2009 Category: Cancer & Oncology Authors: Seon-Hui Shim, Choon-Taek Lee, J. Hun Hah, Jae-Jung Lee, Seok-Woo Park, Dae Seog Heo, Myung-Whun Sung Source Type: journals
Effective relief of neuropathic pain by adeno-associated virus-mediated expression of a small hairpin RNA against GTP cyclohydrolase 1
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Conclusions:
Therefore, the data suggests that GCH1 levels can be reduced by introducing rAAV-shGCH1, leading to pain relief. Based on the results, we propose that GCH1 modulation may be developed as a clinically applicable gene therapy strategy to treat neuropathic pain. (Source: Molecular Pain)
Source: Molecular Pain - November 18, 2009 Category: Molecular Biology Authors: Sung Jin KimWon Il LeeYoon Sun LeeDong Hou KimJin Woo ChangSeong Who KimHeuiran Lee Source Type: journals
The effect of endostatin mediated by human mesenchymal stem cells on ovarian cancer cells in vitro
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Conclusion We found that MSCs possessed great migratory capacity in vitro and the human ovarian adenocarcinoma cell line SKOV3 could
significantly induce the migration of MSCs. Our results provided evidence that MSCs could be utilized as a powerful delivery
system of endostatin. The endostatin produced by MSC-EN cells could inhibit the proliferation of SKOV3 cells.
Content Type Journal ArticleCategory Original PaperDOI 10.1007/s00432-009-0728-8Authors
Jing Jiang, The Second Affiliated Hospital of Harbin Medical University Department of Obstetrics and Gynecology No. 246, Xue Fu Road, Nan Gang District ...
Source: Journal of Cancer Research and Clinical Oncology - November 17, 2009 Category: Cancer & Oncology Tags: Journal of Cancer Research and Clinical Oncology Source Type: journals
T Cell Receptor (TCR) Gene Transfer with Lentiviral Vectors Allows Efficient Redirection of Tumor Specificity in Naive and Memory T Cells Without Prior Stimulation of Endogenous TCR
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Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)
Source: Human Gene Therapy - November 17, 2009 Category: Genetics & Stem Cells Tags: article Source Type: journals
Controllable Expansion of Primary Cardiomyocytes by Reversible Immortalization
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Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)
Source: Human Gene Therapy - November 17, 2009 Category: Genetics & Stem Cells Tags: article Source Type: journals
Therapeutic Vaccination with an Interleukin-2–Interferon-γ-Secreting Allogeneic Tumor Vaccine in Patients with Progressive Castration-Resistant Prostate Cancer: A Phase I/II Trial
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Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)
Source: Human Gene Therapy - November 17, 2009 Category: Genetics & Stem Cells Tags: article Source Type: journals
Researchers To Test First Gene Therapy For Alzheimer's Patients
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Mount Sinai School of Medicine is one of 12 sites nationwide participating in the first Phase 2 clinical trial to test gene therapy treatment for Alzheimer's disease. The study is the first multicenter neurosurgical intervention in Alzheimer's research in the U.S. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - November 17, 2009 Category: Consumer Health News Tags: Alzheimer's / Dementia Source Type: news
Researchers To Test First Gene Therapy For Alzheimer's Patients
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Mount Sinai School of Medicine is one of 12 sites nationwide participating in the first Phase 2 clinical trial to test gene therapy treatment for Alzheimer's disease. The study is the first multicenter neurosurgical intervention in Alzheimer's research in the U.S. The experimental treatment utilizes a viral-based gene transfer system, CERE-110, that makes Nerve Growth Factor (NGF), a naturally occurring protein that helps maintain nerve cell survival in the brain. (Source: Alzheimer's / Dementia News From Medical News Today)
Source: Alzheimer's / Dementia News From Medical News Today - November 17, 2009 Category: Geriatrics Tags: Alzheimer's / Dementia Source Type: news
Mount Sinai researchers to test first gene therapy For Alzheimer's patients
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(The Mount Sinai Hospital / Mount Sinai School of Medicine) Mount Sinai School of Medicine is one of 12 sites nationwide participating in the first Phase 2 clinical trial to test gene therapy treatment for Alzheimer's disease. The study is the first multicenter neurosurgical intervention in Alzheimer's research in the US. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 17, 2009 Category: Global & Universal Source Type: news
Targeted Drug and Gene Delivery Systems for Lung Cancer Therapy.
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CONCLUSIONS: Docetaxel efficacy was enhanced by LHRH receptor-targeted deslorelin conjugate and further improved by combination with targeted antiangiogenic nanoparticle gene therapy. Combination of novel targeted therapeutic approaches described here provides an attractive alternative to the current treatment options for lung cancer therapy. (Clin Cancer Res 2009;15(23):OF1-10).
PMID: 19920099 [PubMed - as supplied by publisher] (Source: Clinical Cancer Research)
Source: Clinical Cancer Research - November 17, 2009 Category: Cancer & Oncology Authors: Sundaram S, Trivedi R, Durairaj C, Ramesh R, Ambati BK, Kompella UB Tags: Clin Cancer Res Source Type: journals
Safety and tolerability of putaminal AADC gene therapy for Parkinson disease
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Conclusion: This study provides class IV evidence that bilateral intrastriatal infusion of adeno-associated viral type 2 vector containing the human AADC gene improves mean scores on the Unified Parkinson’s Disease Rating Scale by approximately 30% in the on and off states, but the surgical procedure may be associated with an increased risk of intracranial hemorrhage and self-limited headache. (Source: Neurology)
Source: Neurology - November 16, 2009 Category: Neurology Authors: Christine, C. W., Starr, P. A., Larson, P. S., Eberling, J. L., Jagust, W. J., Hawkins, R. A., VanBrocklin, H. F., Wright, J. F., Bankiewicz, K. S., Aminoff, M. J. Tags: PET, Parkinson's disease/Parkinsonism, gene therapy ARTICLES Source Type: journals
Amelioration of myocarditis by HVEM-overexpressing dendritic cells through induction of IL-10-producing cells
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Conclusion
Myosin-DC-AdHVEM cell gene therapy appears to be a safe and effective way of inhibiting the development of EAM. The signal induced by HVEM seems to play different roles in different cells. (Source: Cardiovascular Research)
Source: Cardiovascular Research - November 16, 2009 Category: Cardiology Authors: Cai, G., Wang, H., Qin, Q., Zhang, J., Zhu, Z., Liu, M., Shen, Q. Tags: ORIGINAL ARTICLES Source Type: journals
Pharmacological Activation of Guanine Nucleotide Exchange Factors for the Small GTPase Rap1 Recruits High-Affinity β1 Integrins as Coreceptors for Parvovirus B19: Improved Ex Vivo Gene Transfer to Human Erythroid Progenitor Cells
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Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)
Source: Human Gene Therapy - November 16, 2009 Category: Genetics & Stem Cells Tags: article Source Type: journals
Intravitreal Adenoviral 15-Lipoxygenase-1 Gene Transfer Prevents Vascular Endothelial Growth Factor A-Induced Neovascularization in Rabbit Eyes
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Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)
Source: Human Gene Therapy - November 16, 2009 Category: Genetics & Stem Cells Tags: article Source Type: journals
Ex Vivo Serotype-Specific Transduction of Equine Joint Tissue by Self-Complementary Adeno-Associated Viral Vectors
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Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)
Source: Human Gene Therapy - November 16, 2009 Category: Genetics & Stem Cells Tags: article Source Type: journals
Activation of Human Dendritic Cells by Recombinant Modified Vaccinia Virus Ankara Vectors Encoding Survivin and IL-2 Genes In Vitro
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Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)
Source: Human Gene Therapy - November 16, 2009 Category: Genetics & Stem Cells Tags: article Source Type: journals
Stable Transgene Expression in Primitive Human CD34+ Hematopoietic Stem/Progenitor Cells, Using the Sleeping Beauty Transposon System
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Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)
Source: Human Gene Therapy - November 16, 2009 Category: Genetics & Stem Cells Tags: article Source Type: journals
Molecularly Evolved Thymidylate Synthase Inhibits 5-Fluorodeoxyuridine Toxicity in Human Hematopoietic Cells
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Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)
Source: Human Gene Therapy - November 16, 2009 Category: Genetics & Stem Cells Tags: article Source Type: journals
Research Sheds Light on Causes of Parkinson's
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Gene mutations linked to both common and rarer hereditary forms of disease Source: HealthDay
Related MedlinePlus Topics: Genes and Gene Therapy, Parkinson's Disease (Source: MedlinePlus Health News)
Source: MedlinePlus Health News - November 15, 2009 Category: Consumer Health News Source Type: consumer
Homing endonucleases: from basics to therapeutic applications.
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Homing endonucleases (HE) are double-stranded DNAses that target large recognition sites (12-40 bp). HE-encoding sequences are usually embedded in either introns or inteins. Their recognition sites are extremely rare, with none or only a few of these sites present in a mammalian-sized genome. However, these enzymes, unlike standard restriction endonucleases, tolerate some sequence degeneracy within their recognition sequence. Several members of this enzyme family have been used as templates to engineer tools to cleave DNA sequences that differ from their original wild-type targets. These custom HEs can be used to stimu...
Source: Cellular and Molecular Life Sciences : CMLS - November 15, 2009 Category: Cytology Authors: Marcaida MJ, Muñoz IG, Blanco FJ, Prieto J, Montoya G Tags: Cell Mol Life Sci Source Type: journals
Gene therapy can improve muscle mass and strength in monkeys, research suggests
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Scientists are one step closer to clinical trials to test a gene delivery strategy to improve muscle mass and function in patients with certain degenerative muscle disorders. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - November 14, 2009 Category: Science Source Type: news
Gene Therapy Can Improves Muscle Mass And Strength In Monkeys, Research Suggests
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Scientists are one step closer to clinical trials to test a gene delivery strategy to improve muscle mass and function in patients with certain degenerative muscle disorders. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - November 14, 2009 Category: Science Source Type: news
Neuroprotective effect of combined hypoxia-induced VEGF and bone marrow-derived mesenchymal stem cell treatment
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Conclusions These results suggest that combined pEpo-SV-VEGF and BMSC treatment is effective in protecting neurons against hypoxic ischemic
injury.
Content Type Journal ArticleCategory Original PaperDOI 10.1007/s00381-009-1040-2Authors
Sung Su An, Yonsei University Department of Neurosurgery, Spine & Spinal Cord Institute, College of Medicine Seoul 120-752 South KoreaHong Lian Jin, Yonsei University Department of Neurosurgery, Spine & Spinal Cord Institute, College of Medicine Seoul 120-752 South KoreaKeung Nyun Kim, Yonsei University Department of Neurosurgery, Spine & Spinal Cord Institute, College o...
Source: Child's Nervous System - November 14, 2009 Category: Pediatrics Tags: Child's Nervous System Source Type: journals
Morphological Changes Induced by Insulin-Like Growth Factor-I Gene Therapy in Pituitary Cell Populations in Experimental Prolactinomas
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Cells Tissues Organs (DOI:10.1159/000258701) (Source: Cells Tissues Organs)
Source: Cells Tissues Organs - November 13, 2009 Category: Cytology Source Type: journals
Alternative-splicing-based bicistronic vectors for ratio-controlled protein expression and application to recombinant antibody production
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In the last decade polycistronic vectors have become essential tools for both basic science and gene therapy applications. In order to co-express heterologous polypeptides, different systems have been developed from Internal Ribosome Entry Site (IRES) based vectors to the use of the 2A peptide. Unfortunately, these methods are not fully suitable for the efficient and reproducible modulation of the ratio between the proteins of interest. Here we describe a novel bicistronic vector type based on the use of alternative splicing. By modifying the consensus sequence that governs splicing, we demonstrate that the ratio between t...
Source: Nucleic Acids Research - November 13, 2009 Category: Research Authors: Fallot, S., Ben Naya, R., Hieblot, C., Mondon, P., Lacazette, E., Bouayadi, K., Kharrat, A., Touriol, C., Prats, H. Tags: Recombinant DNA expression, Ribosomes and Protein Translation Methods Online Source Type: journals
Weak Link In Cancer Cell Armor Identified
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The seeming invincibility of cancerous tumors may be crumbling, thanks to a promising new gene therapy that eliminates the ability of certain cells to repair themselves. Researchers have discovered that inactivation of a DNA repair gene called Hus1 efficiently kills cells lacking p53 -- a gene mutated in the majority of human cancers. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - November 13, 2009 Category: Science Source Type: news
Researchers Identify A Weak Link In Cancer Cell Armor
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The seeming invincibility of cancerous tumors may be crumbling, thanks to a promising new gene therapy that eliminates the ability of certain cells to repair themselves. Researchers at the Cornell University College of Veterinary Medicine have discovered that inactivation of a DNA repair gene called Hus1 efficiently kills cells lacking p53 -- a gene mutated in the majority of human cancers. (Source: Cancer / Oncology News From Medical News Today)
Source: Cancer / Oncology News From Medical News Today - November 13, 2009 Category: Cancer & Oncology Tags: Cancer / Oncology Source Type: news
Researchers Identify A Weak Link In Cancer Cell Armor
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The seeming invincibility of cancerous tumors may be crumbling, thanks to a promising new gene therapy that eliminates the ability of certain cells to repair themselves. Researchers at the Cornell University College of Veterinary Medicine have discovered that inactivation of a DNA repair gene called Hus1 efficiently kills cells lacking p53 -- a gene mutated in the majority of human cancers. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - November 13, 2009 Category: Consumer Health News Tags: Cancer / Oncology Source Type: news
Safety Evaluation of AAV2-GDNF Gene Transfer into the Dopaminergic Nigrostriatal Pathway in Aged and Parkinsonian Rhesus Monkeys
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Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)
Source: Human Gene Therapy - November 12, 2009 Category: Genetics & Stem Cells Tags: article Source Type: journals
Nonintegrating Lentiviral Vectors Can Effectively Deliver Ovalbumin Antigen for Induction of Antitumor Immunity
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Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)
Source: Human Gene Therapy - November 12, 2009 Category: Genetics & Stem Cells Tags: article Source Type: journals
Anti-CD3 antibodies modulate anti-factor VIII immune responses in hemophilia A mice after factor VIII plasmid-mediated gene therapy
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One major obstacle in gene therapy is the generation of immune responses directed against transgene product. Five consecutive anti-CD3 treatments concomitant with factor VIII (FVIII) plasmid injection prevented the formation of inhibitory antibodies against FVIII and achieved persistent, therapeutic levels of FVIII gene expression in treated hemophilia A mice. Repeated plasmid gene transfer is applicable in tolerized mice without eliciting immune responses. Anti-CD3 treatment significantly depleted both CD4+ and CD8+ T cells, whereas increased transforming growth factor-β levels in plasma and the frequency of both CD4...
Source: Blood - November 12, 2009 Category: Hematology Authors: Peng, B., Ye, P., Rawlings, D. J., Ochs, H. D., Miao, C. H. Tags: Thrombosis and Hemostasis, gene therapy Source Type: journals
MDA Grantees Prove Gene Therapy Grows Muscle Strength & Size In Primates
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Four macaque monkeys that received injections of genes for a protein called follistatin into upper leg muscles experienced pronounced and durable increases in muscle size and strength and no adverse effects, the Muscular Dystrophy Association (MDA) announced. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - November 12, 2009 Category: Consumer Health News Tags: Muscular Dystrophy / ALS Source Type: news
MDA Grantees Prove Gene Therapy Grows Muscle Strength & Size In Primates
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Four macaque monkeys that received injections of genes for a protein called follistatin into upper leg muscles experienced pronounced and durable increases in muscle size and strength and no adverse effects, the Muscular Dystrophy Association (MDA) announced. The findings could have implications particularly for injured and aging people worldwide; and for tens of millions experiencing muscle loss associated with cancer, AIDs and muscle diseases. (Source: Muscular Dystrophy News From Medical News Today)
Source: Muscular Dystrophy News From Medical News Today - November 12, 2009 Category: Neurology Tags: Muscular Dystrophy / ALS Source Type: news
Cornell researchers identify a weak link in cancer cell armor
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(Cornell University) The seeming invincibility of cancerous tumors may be crumbling, thanks to a promising new gene therapy that eliminates the ability of certain cells to repair themselves. Researchers at the Cornell University College of Veterinary Medicine have discovered that inactivation of a DNA repair gene called Hus1 efficiently kills cells lacking p53 -- a gene mutated in the majority of human cancers. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 12, 2009 Category: Global & Universal Source Type: news
Q&A: Gene therapy turnaround?
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Geneticist Mark Kay discusses the field's recent progress and the hurdles still left to overcome (Source: The Scientist)
Source: The Scientist - November 12, 2009 Category: Science Authors: Victoria Stern Source Type: info
Success Boosting Monkey Muscle Could Help Humans
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Researchers have successfully used gene therapy to increase monkeys' muscle strength. The team hopes to use the same treatment to help people with muscle-wasting diseases grow back their muscle strength.» E-Mail This » Add to Del.icio.us (Source: NPR Health and Science)
Source: NPR Health and Science - November 11, 2009 Category: Consumer Health News Source Type: news
Boys with ALD bring gene therapy in from cold
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Two boys treated with a gene therapy for the brain disease X-linked adrenoleukodystrophy have fared so well that doctors are seeking more volunteers (Source: New Scientist - Health)
Source: New Scientist - Health - November 11, 2009 Category: Consumer Health News Source Type: journals
Boys with ALD bring gene therapy in from cold
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Two boys treated with a gene therapy for the brain disease X-linked adrenoleukodystrophy have fared so well that doctors are seeking more volunteers (Source: New Scientist - Genetics)
Source: New Scientist - Genetics - November 11, 2009 Category: Genetics & Stem Cells Source Type: journals
Gene therapy: Nerve repair
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Nature 462, 140 (2009). doi:10.1038/462140a (Source: Nature)
Source: Nature - November 11, 2009 Category: Research Tags: Research Highlights Source Type: journals
Homozygous familial hypercholesterolemia: Long term clinical course and plasma exchange therapy for two individual patients and review of the literature
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Familial hypercholesterolemia (FH) is an autosomal dominant disease. Homozygous FH (HFH) manifests with severe hypercholesterolemia since birth (cholesterol levels >5-6 the upper normal limit), which, if untreated, leads to early onset accelerated atherosclerosis and premature coronary death, usually before the 2nd or 3rd decades of life. Various invasive procedures (iliocecal bypass, porto-caval shunt, liver transplant, and gene therapy) have been introduced for lowering low density lipoprotein (LDL) aiming at reducing atherosclerosis and improving survival of HFH patients. Of all the various methods, LDL apheresis has be...
Source: Journal of Clinical Apheresis - November 10, 2009 Category: Hematology Authors: Roy Beigel, Yitzhak Beigel Source Type: journals
