Targeted gene editing enters clinic

March 2, 2011

Patients with HIV first to receive experimental gene therapy. A gene-therapy method that specifically disrupts a single gene may have had its first success in the clinic, potentially boosting immune-cell counts in a small number of patients with HIV. The results, presented on 28 February at the Conference on Retroviruses and Opportunistic Infections in Boston, Massachusetts, mark an important therapeutic test for enzymes known as zinc finger nucleases — small proteins that can be designed to bind to and edit specific DNA sequences by virtue of their zinc-bearing structures. (Nature News)