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- W2017011262 abstract "Duchenne muscular dystrophy (DMD) is a lethal genetic muscle disorder affecting 1:3500 male individuals, caused by recessive mutations in the dystrophin gene. The size of the gene (2.4Mb) and mRNA (14kb) in addition to immunogenicity problems and inefficient transduction of mature myofibres by currently available vector systems are formidable obstacles to the development of gene therapy. AAV vectors overcome many of the problems associated with other vectors but accommodate limited transgene capacity (<5kb), allowing only truncated versions of dystrophin (microdystrophin) to be packaged." @default.
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- W2017011262 date "2006-01-01" @default.
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- W2017011262 title "907. Development of Recombinant Novel Adeno-Associated Viral (rAAV) Vectors Encoding Optimised Microdystrophin cDNAs for Duchenne Muscular Dystrophy (DMD)" @default.
- W2017011262 doi "https://doi.org/10.1016/j.ymthe.2006.08.997" @default.
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