Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapy

Despite significant advances in cystic fibrosis (CF) treatments, a one-time treatment for this life-shortening disease remains elusive. Stable complementation of the disease-causing mutation with a normal copy of the CF transmembrane conductance regulator (CFTR) gene fulfills that goal. Integrating...

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Bibliografiske detaljer
Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Marquez Loza, Laura I., Cooney, Ashley L., Dong, Qian, Randak, Christoph O., Rivella, Stefano, Sinn, Patrick L., McCray, Paul B.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2021
Fag:
Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7973238/
https://ncbi.nlm.nih.gov/pubmed/33768133
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2021.02.020
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