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Gene Therapy of mdx Mice With Large Truncated Dystrophins Generated by Recombination Using rAAV6

Recombinant adeno-associated viral (rAAV) vector-mediated gene transfer represents a promising approach for many diseases. However, the applicability of rAAV vectors has long been hindered by the small (~4.8 kb) DNA packaging capacity. This limitation can hamper the packaging and delivery of critica...

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Autori principali: Odom, Guy L, Gregorevic, Paul, Allen, James M, Chamberlain, Jeffrey S
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2011
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3017440/
https://ncbi.nlm.nih.gov/pubmed/20859263
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.205
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