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

Mol Ther Methods Clin Dev. 2021-02; 
Laura I Marquez Loza, Ashley L Cooney, Qian Dong, Christoph O Randak, Stefano Rivella, Patrick L Sinn, Paul B McCray
Products/Services Used Details Operation
PCR and Cloning A proprietary algorithm (GenScript) was used for coCFTR2 Get A Quote

摘要

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 () 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 codon optimization (co)... More

关键词

CFTR, Cystic fibrosis, codon optimization, gene therapy, lentiviral vectors