A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence

Recombinant adeno-associated virus (rAAV) is currently the best vector for gene delivery into the skeletal muscle. However, the 5-kb packaging size of this virus is a major obstacle for large gene transfer. This past decade, many different strategies were developed to circumvent this issue (concatem...

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Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Pryadkina, Marina, Lostal, William, Bourg, Nathalie, Charton, Karine, Roudaut, Carinne, Hirsch, Matthew L, Richard, Isabelle
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group 2015
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4445010/
https://ncbi.nlm.nih.gov/pubmed/26029720
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2015.9
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