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

ABSTRACT: 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 lentiviral vectors are well suited for this purpose, but widespread airway transduction in humans is limited by achievable titers and delivery barriers. Since airway epithelial cells are interconnected through gap junctions, small numbers of cells expressing supraphysiologic levels of CFTR could support sufficient channel function to rescue CF phenotypes. Here, we investigated promoter choice and CFTR codon optimization (coCFTR) as strategies to regulate CFTR expressi

SUBMITTER: Marquez Loza LI 

PROVIDER: S-EPMC7973238 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature