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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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Udgivet i:Proc Natl Acad Sci U S A
Principais autores: Ostedgaard, Lynda S., Zabner, Joseph, Vermeer, Daniel W., Rokhlina, Tatiana, Karp, Philip H., Stecenko, Arlene A., Randak, Christoph, Welsh, Michael J.
Format: Artigo
Sprog:Inglês
Udgivet: National Academy of Sciences 2002
Fag:
Biological Sciences
Online adgang: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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