CFTR with a partially deleted R domain corrects the cystic fibrosis chloride transport defect in human airway epithelia in vitro and in mouse nasal mucosa in vivo

In developing gene therapy for cystic fibrosis (CF) airways disease, a transgene encoding a partially deleted CF transmembrane conductance regulator (CFTR) Cl(−) channel could be of value for vectors such as adeno-associated virus that have a limited packaging capacity. Earlier studies in heterologo...

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Pubblicato in:	Proc Natl Acad Sci U S A
Autori principali:	Ostedgaard, Lynda S., Zabner, Joseph, Vermeer, Daniel W., Rokhlina, Tatiana, Karp, Philip H., Stecenko, Arlene A., Randak, Christoph, Welsh, Michael J.
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	National Academy of Sciences 2002
Soggetti:	Biological Sciences
Accesso online:	https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC122478/ https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/11854474/ https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.261714599
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