Breakthrough gene therapy could preserve vision
Washington, Aug 17 : Non-viral gene therapy can delay the onset of some forms of eye disease and preserve vision, found one of only two studies of its kind.
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Breakthrough gene therapy prevents retinal degeneration
( Tufts University, Health Sciences ) In one of only two studies of its kind, a study from researchers at Tufts University demonstrates that non-viral gene therapy can delay the onset of some forms of eye disease and preserve vision. The team developed nanoparticles to deliver therapeutic genes to the retina and found that treated mice temporarily retained more eyesight than controls. The study …
Gene Therapy To Treat Diseases Of The Eye
The easy accessibility of the eye and the established link between specific genetic defects and ocular disorders offer hope for using gene therapy to provide long-term therapeutic benefit. Two reports in the current issue of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc., describe the effective replacement of a human gene to preserve photoreceptor function in a mouse model of severe retinal degeneration. The articles are available free online here… (Source: Health News from Medical News Today)
Effects of Antisense Transforming Growth Factor-beta1 Gene Transfer on the Biological Activities of Tendon Sheath Fibroblasts.
Authors: Xia C, Ding C, Yang X, Sun K, Tian S
Recent studies have shown the importance of transforming growth factor-beta (TGF-beta) in flexor tendon wound healing. Decreased adhesion formation and increased range of motion after the administration of TGF-beta antibodies after tendon repair have been shown. But TGF-beta antibodies have a short biologic half-life, and continuous supplementation of exogenous TGF-beta antibodies is not practical. Transfer of growth factor genes to tenocytes provides an alternative to protein therapeutics, and a gene therapy approach will prolong the availability of therapeutic proteins.We investigated the biological activities effects of rabbit tendon sheath fibroblasts transfected by antisense TGF-beta1 gene. Tendon sheath fibroblasts were isolated from …
Non-viral gene therapy delays onset of eye disease
In one of only two studies of its kind, a study from researchers at Tufts University School of Medicine and the Sackler School of Graduate Biomedical Sciences at Tufts demonstrates that non-viral gene therapy can delay the onset of some forms of eye disease and preserve vision. The team developed nanoparticles to deliver therapeutic genes to the retina and found that treated mice temporarily …
Study finds no link among estrogen and lung cancer mortality in postmenopausal women
Use of estrogen alone did not increase lung cancer mortality in postmenopausal women, according to a study published online August 13 in The Journal of the National Cancer Institute. In the Women’s Health Initiative (WHI) trial, which consisted of several clinical trials on postmenopausal women, one study showed women with previous hysterectomy taking combined estrogen plus progestin therapy had …
Scientists discover new signaling pathway to reduce tumour growth in glioblastoma multiforme
Malignant gliomas are the most common subtype of primary brain tumor – and one of the deadliest. Even as doctors make steady progress treating other types of solid tumor cancers, from breast to prostate, the most aggressive form of malignant glioma, called a glioblastoma multiforme or GBM, has steadfastly defied advances in neurosurgery, radiation therapy and various conventional or novel drugs …
Scientists discover genetic alteration in Kabuki syndrome
Using a new, rapid and less expensive DNA sequencing strategy, scientists have discovered genetic alterations that account for most cases of Kabuki syndrome, a rare disorder that causes multiple birth defects and mental retardation. Instead of sequencing the entire human genome, the new approach sequences just the exome, the 1-2 percent of the human genome that contains protein-coding genes.
Common genetic variation in HLA region associated with late onset sporadic Parkinson’s disease: Research
A team of researchers has discovered new evidence that Parkinson’s disease may have an infectious or autoimmune origin. “Common genetic variation in the HLA region is associated with late-onset sporadic Parkinson’s disease” appears online in Nature Genetics.
Bay Area families lead research efforts for Rett Syndrome
Rett syndrome is one of thousands of genetic disorders in existence — but one of the few many people believe will soon be reversible. Scientists were able to reverse symptoms of the neurodevelopmental disorder in mice in 2007, giving hope to families everywhere.
Enhanced Long-Term Transduction and Multilineage Engraftment of Human Hematopoietic Stem Cells Transduced with Tyrosine-Modified Recombinant Adeno-Associated Virus Serotype 2
Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)
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Study finds Olaparib can reduce tumor size in ovarian cancer women
Understanding the underlying genetic weakness of certain types of cancer may lead to targeted therapy and provide the key to effective treatment, a new study suggests. An international consortium of researchers has shown that an investigational drug, Olaparib, can reduce the size of tumors in women with advanced hereditary ovarian cancer with BRCA gene mutations.
Gene Therapy For Brain Cancer Could Be Improved By Deathstalker Scorpion Venom
An ingredient in the venom of the “deathstalker” scorpion could help gene therapy become an effective treatment for brain cancer, scientists are reporting. The substance allows therapeutic genes – genes that treat disease – to reach more brain cancer cells than current approaches, according to the study in ACS Nano, a monthly journal. Miqin Zhang and colleagues note that gene therapy – the delivery of therapeutic genes into diseased cells – shows promise for fighting glioma, the most common and most serious form of brain cancer… (Source: Health News from Medical News Today)
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A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells
To develop safer and more effective vectors for gene therapy of X-linked severe combined immunodeficiency (SCID-X1), we have evaluated new self-inactivating lentiviral vectors based on the HIV virus. The CL20i4-hc-Revgen vector contains the entire human common chain (c) genomic sequence driven by the c promoter. The CL20i4-EF1-hcOPT vector uses a promoter fragment from the eukaryotic elongation factor alpha (EF1) gene to express a codon-optimized human c cDNA. Both vectors contain a 400-bp insulator fragment from the chicken β-globin locus within the self-inactivating long-terminal repeat. Transduction of bone marrow cells using either of these vectors restored T, B, and natural killer lymphocyte development and function in a mouse SCID-X1 transplantation model. Transduction of human …
The combination of immunosuppression and carrier cells significantly enhances the efficacy of oncolytic poxvirus in the pre-immunized host
Authors: Z S Guo, V Parimi, M E O’Malley, P Thirunavukarasu, M Sathaiah, F Austin
& D L Bartlett (Source: Gene Therapy)
Robust cardiomyocyte-specific gene expression following systemic injection of AAV: in vivo gene delivery follows a Poisson distribution
Authors: K-M R Prasad, Y Xu, Z Yang, S T Acton
& B A French (Source: Gene Therapy)
Combinatorial gene therapy induces regression of hepatic encephalopathy
Authors: F J Gálvez-Gastélum, J J Garcia-Bañuelos, C Beas-Zárate, A Segura-Flores, H González, V Chaparro-Huerta, A Salazar-Montes, A S Sandoval-Rodriguez, M Bueno-Topete, S Lucano-Landeros, D Medina-Preciado, I Gonzalez-Garcia
& J Armendáriz-Borunda (Source: Gene Therapy)
Gene therapy in the second eye of RPE65-deficient dogs improves retinal function
Authors: M J Annear, J T Bartoe, S E Barker, A J Smith, P G Curran, J W Bainbridge, R R Ali
& S M Petersen-Jones (Source: Gene Therapy)
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Systemic delivery of E6/7 siRNA using novel lipidic particles and its application with cisplatin in cervical cancer mouse models
Systemic delivery of E6/7 siRNA using novel lipidic particles and its application with cisplatin in cervical cancer mouse models
Gene Therapy advance online publication, August 12, 2010. doi:10.1038/gt.2010.113
Authors: S Y Wu, A Singhania, M Burgess, L N Putral, C Kirkpatrick, N M Davies
& N A J McMillan (Source: Gene Therapy)
Promising results of gene therapy to treat diseases of the eye
The easy accessibility of the eye and the established link between specific genetic defects and ocular disorders offer hope for using gene therapy to provide long-term therapeutic benefit. Two reports in the current issue of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc., describe the effective replacement of a human gene to preserve photoreceptor function in a …
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