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Functional Cystic Fibrosis Transmembrane Conductance Regulator Expression in Cystic Fibrosis Airway Epithelial Cells by AAV6.2-Mediated Segmental Trans-Splicing

Cystic fibrosis is characterized by deficiency of the cystic fibrosis transmembrane conductance regulator (CFTR), a Cl(−) transporter. The packaging constraints of adeno-associated viral (AAV) vectors preclude delivery of both an active promoter and CFTR cDNA to target cells. We hypothesized that se...

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Hlavní autoři: Song, Yuhu, Lou, Howard H., Boyer, Julie L., Limberis, Maria P., Vandenberghe, Luk H., Hackett, Neil R., Leopold, Philip L., Wilson, James M., Crystal, Ronald G.
Médium: Artigo
Jazyk:Inglês
Vydáno: Mary Ann Liebert, Inc. 2009
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC2855253/
https://ncbi.nlm.nih.gov/pubmed/19257851
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2008.173
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