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

Abstract excerpt

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...

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