CAREER: Engineering Recombinant Lentiviruses for Cystic Fibrosis Gene Therapy

Georgia Tech Research Corporation, Atlanta GA

Investigators

Abstract

0134409 Le Doux The objective of the proposed research is to develop greater understanding of lentivirus-cell interactions, with the long-range goal of developing improved lentivirues that will be useful for cystic fibrosis gene therapy. Specifically, the goal of this research is to develop a targeted lentiviral vector and to examine the intracellular pathway that leads to maximal transfection. The specific tasks of this research include: (1) the test of the hypothesis that lentivirus gene transfer can be improved by incorporating escort proteins into the lipid bilayer of the viruses, (2) the quantitative analysis of the effect of the escort proteins on infection, and (3) the engineering of lentiviruses to target receptors on the apical surfaces of polarized epithelial cells. The knowledge gained from this research could help the design of the next generation of lentivirus vectors that would be useful for human gene therapy protocols.