Cystic fibrosis transmembrane conductance regulator with a shortened R domain rescues the intestinal phenotype of CFTR(−/−) mice

Gene transfer could provide a novel therapeutic approach for cystic fibrosis (CF), and adeno-associated virus (AAV) is a promising vector. However, the packaging capacity of AAV limits inclusion of the full-length cystic fibrosis transmembrane conductance regulator (CFTR) cDNA together with other re...

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Detalhes bibliográficos
Main Authors: Ostedgaard, Lynda S., Meyerholz, David K., Vermeer, Daniel W., Karp, Philip H., Schneider, Lindsey, Sigmund, Curt D., Welsh, Michael J.
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2011
Assuntos:
Biological Sciences
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3041073/
https://ncbi.nlm.nih.gov/pubmed/21285372
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1019752108
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