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

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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Bibliographic Details
Main Authors: Laura I. Marquez Loza, Ashley L. Cooney, Qian Dong, Christoph O. Randak, Stefano Rivella, Patrick L. Sinn, Paul B. McCray, Jr.
Format: Article
Language:English
Published: Elsevier 2021-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Cystic fibrosis
lentiviral vectors
gene therapy
codon optimization
CFTR
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121000358

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