Archives de la catégorie gene therapy

Les tests génétiques prédisent-ils notre avenir ?

Science publique, par Michel Alberganti, le 06.05.2011 à 14:00

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Notch1 in Bone Marrow–Derived Cells Mediates Cardiac Repair After Myocardial Infarction

Notch1 in Bone Marrow–Derived Cells Mediates Cardiac Repair After Myocardial Infarction

Yuxin Li, Yukio Hiroi, Soeun Ngoy, BS; Ryuji Okamoto, Kensuke Noma, Chao-Yung Wang, MD; Hong-Wei Wang, Qian Zhou, MD; Freddy Radtke, Ronglih Liao, PhD James K. Liao

Circulation. 2011 Published online before print February 14, 2011, doi: 10.1161/CIRCULATIONAHA.110.947531

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Controlling transgene expression in subcutaneous implants using a skin lotion containing the apple metabolite phloretin

Controlling transgene expression in subcutaneous implants using a skin lotion containing the apple metabolite phloretin

Marc Gitzinger, Christian Kemmer, Marie Daoud El-Baba, Wilfried Webera, and Martin Fussenegger

Published online before print June 22, 2009, doi: 10.1073/pnas.0901501106

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Gene therapy success ‘reverses’ blindness

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Tiny Magnets Offer Breakthrough In Gene Therapy For Cancer

Tiny Magnets Offer Breakthrough In Gene Therapy For Cancer: (Apr. 21, 2008) — A revolutionary cancer treatment using microscopic magnets to enable ‘armed’ human cells to target tumours has been developed. A new study shows that inserting these nanomagnets into cells carrying genes to fight tumours, results in many more cells successfully reaching and invading malignant tumours. »

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Deadly Genetic Disease Prevented Before Birth In Zebrafish

Deadly Genetic Disease Prevented Before Birth In Zebrafish: « (Mar. 21, 2008) — By injecting a customized ‘genetic patch’ into early stage fish embryos, researchers at Washington University School of Medicine in St. Louis were able to correct a genetic mutation so the embryos developed normally. »


In vivo correction of a Menkes disease model using antisense oligonucleotides, Erik C. Madsen, Paul A. Morcos, Bryce A. Mendelsohn, and Jonathan D. Gitlin

Although the molecular basis of many inherited metabolic diseases has been defined, the availability of effective therapies in such disorders remains problematic. Menkes disease is a fatal neurodegenerative disorder due to loss-of-function mutations in the ATP7A gene encoding a copper-transporting P-type Atpase. To develop therapeutic approaches in affected patients, we have identified a zebrafish model of Menkes disease termed calamity that results from splicing defects in the zebrafish orthologue of the ATP7A gene. Embryonic-recessive lethal mutants have impaired copper homeostasis that results in absent melanin pigmentation, impaired notochord formation, and hindbrain neurodegeneration. In this current study, we have attempted to rescue these striking phenotypic alterations by using a series of antisense morpholino oligonucleotides directed against the splice-site junctions of two mutant calamity alleles. Our findings reveal a robust and complete correction of the copper-deficient defects of calamity in association with the generation of the WT Menkes protein in all rescued mutants. Interestingly, a quantitative analysis of atp7a-specific transcripts suggests that competitive translational regulation may account for the synthesis of WT protein in these embryos. This in vivo correction of Menkes disease through the rescue of aberrant splicing may provide therapeutic options in this fatal disease and illustrates the potential for zebrafish models of human genetic disease in the development of treatments based on the principles of interactions of synthetic oligonucleotide analogues with mRNA.

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Gene Therapy ‘Trains’ Immune System To Destroy Brain Cancer Cells And Reverses Behavioral Deficits

ScienceDaily (Feb. 22, 2008) — A new gene therapy approach that attracts and « trains » immune system cells to destroy deadly brain cancer cells also provides long-term immunity, produces no significant adverse effects and — in the process of destroying the tumor — promotes the return of normal brain function and behavioral skills, according to a study conducted by researchers at Cedars-Sinai Medical Center’s Board of Governors Gene Therapeutics Research Institute.

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