Transfer of the Full-Length Dystrophin-Coding Sequence into Muscle Cells by a Dual High-Capacity Hybrid Viral Vector with Site-Specific Integration Ability

Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene, making it a potential target for gene therapy. There is, however, a scarcity of vectors that can accommodate the 14-kb DMD cDNA and permanently genetically correct muscle tissue in vivo or proliferating myogenic progenitors in...

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Main Authors: Gonçalves, Manuel A. F. V., van Nierop, Gijsbert P., Tijssen, Marloes R., Lefesvre, Pierre, Knaän-Shanzer, Shoshan, van der Velde, Ietje, van Bekkum, Dirk W., Valerio, Dinko, de Vries, Antoine A. F.
Format: Artigo
Sprog:Inglês
Udgivet: American Society for Microbiology 2005
Fag:
Gene Delivery
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC548431/
https://ncbi.nlm.nih.gov/pubmed/15709034
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1128/JVI.79.5.3146-3162.2005
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