Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1

Adeno-associated viral (AAV) vectors (serotype 2) efficiently transduce skeletal muscle, and have been used as gene delivery vehicles for hemophilia B and for muscular dystrophies in experimental animals and humans. Recent reports suggest that AAV vectors based on serotypes 1, 5, and 7 transduce mur...

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Publicado en:Blood
Autores principales: Arruda, Valder R., Schuettrumpf, Joerg, Herzog, Roland W., Nichols, Timothy C., Robinson, Nancy, Lotfi, Yasmin, Mingozzi, Federico, Xiao, Weidong, Couto, Linda B., High, Katherine A.
Formato: Artigo
Lenguaje:Inglês
Publicado: 2003
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC4704086/
https://ncbi.nlm.nih.gov/pubmed/12969984
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2003-05-1446
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