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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Publié dans:	J Virol
Auteurs principaux:	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
Langue:	Inglês
Publié:	American Society for Microbiology (ASM) 2005
Sujets:	Gene Delivery
Accès en ligne:	https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC548431/ https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/15709034/ https://ncbi.nlm.nih.govhttps://doi.org/10.1128/JVI.79.5.3146-3162.2005
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