Gel-mediated Delivery of AAV1 Vectors Corrects Ventilatory Function in Pompe Mice With Established Disease

Pompe disease is a muscular dystrophy that results in respiratory insufficiency. We characterized the outcomes of targeted delivery of recombinant adeno-associated virus serotype 1 (rAAV2/1) vector to diaphragms of Pompe mice with varying stages of disease progression. We observed significant improv...

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Detalhes bibliográficos
Main Authors: Mah, Cathryn S, Falk, Darin J, Germain, Sean A, Kelley, Jeffry S, Lewis, Melissa A, Cloutier, Denise A, DeRuisseau, Lara R, Conlon, Thomas J, Cresawn, Kerry O, Fraites Jr, Thomas J, Campbell-Thompson, Martha, Fuller, David D, Byrne, Barry J
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2010
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Original Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2839425/
https://ncbi.nlm.nih.gov/pubmed/20104213
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.305
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