GENE EDITING FOR HIV GENE THERAPY

摘要

The present disclosure is in the field of genome engineering, particularly targeted integration of anti-HIV transgenes into the genome of a cell for the treatment and/or prevention of HIV.

主权项

1. A method of generating a cell that will not sustain HIV infection, the method comprising genetically modifying the cell such that:
(i) an endogenous restriction factor is modified to increase its activity against HIV; (ii) an endogenous viral host factor gene is modified to decrease its ability to support HIV infection; or (iii) an exogenous transgene encoding a Trim5α-Cyp fusion protein is inserted into an endogenous safe harbor locus wherein the cell will not sustain HIV infection.