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Researchers from University of California School of Medicine Discuss Findings in Gene Therapy (Retinal gene therapy with a large MYO7A cDNA using...

July 3, 2014



Researchers from University of California School of Medicine Discuss Findings in Gene Therapy (Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus)

By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Current study results on Biotechnology have been published. According to news reporting from Los Angeles, California, by NewsRx journalists, research stated, "Usher 1 patients are born profoundly deaf and then develop retinal degeneration. Thus they are readily identified before the onset of retinal degeneration, making gene therapy a viable strategy to prevent their blindness."

The news correspondents obtained a quote from the research from the University of California School of Medicine, "Here, we have investigated the use of adeno-associated viruses (AAVs) for the delivery of the Usher 1B gene, MYO7A, to retinal cells in cell culture and in Myo7a-null mice. MYO7A cDNA, under control of a smCBA promoter, was packaged in single AAV2 and AAV5 vectors and as two overlapping halves in dual AAV2 vectors. The 7.9-kb smCBA-MYO7A exceeds the capacity of an AAV vector; packaging of such oversized constructs into single AAV vectors may involve fragmentation of the gene. Nevertheless, the AAV2 and AAV5 single vector preparations successfully transduced photoreceptor and retinal pigment epithelium cells, resulting in functional, full-length MYO7A protein and correction of mutant phenotypes, suggesting successful homologous recombination of gene fragments. With discrete, conventional-sized dual AAV2 vectors, full-length MYO7A was detected, but the level of protein expression was variable, and only a minority of cells showed phenotype correction."

According to the news reporters, the research concluded: "Our results show that MYO7A therapy with AAV2 or AAV5 single vectors is efficacious; however, the dual AAV2 approach proved to be less effective."

For more information on this research see: Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus. Gene Therapy, 2013;20(8):824-33. (Nature Publishing Group - www.nature.com/; Gene Therapy - www.nature.com/gt/)

Our news journalists report that additional information may be obtained by contacting V.S. Lopes, Departments of Ophthalmology and Neurobiology, Jules Stein Eye Institute, UCLA School of Medicine, Los Angeles, CA 90095, United States. Additional authors for this research include S.E. Boye, C.M. Louie, S. Boye, F. Dyka, V. Chiodo, H. Fofo, W.W. Hauswirth and D.S Williams (see also Biotechnology).

Keywords for this news article include: Biotechnology, Therapy, California, Los Angeles, United States, North and Central America.

Our reports deliver fact-based news of research and discoveries from around the world. Copyright 2014, NewsRx LLC


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Source: Gene Therapy Weekly


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