一段话概述
Long before CRISPR, Sangamo was editing human genes with zinc finger nucleases — and running the first human gene-editing trials, starting in 2009 with an HIV programme. Today it works on zinc-finger repressors for neurological disease and on the delivery capsids that get genetic medicines into the brain.
Sangamo Therapeutics, founded in 1995, pioneered zinc finger nuclease therapeutics and conducted the first human gene-editing trials, including CCR5 disruption in HIV from 2009. Current work centres on zinc finger repressors for neurological targets such as prion disease and tauopathies, and on engineered AAV capsids for central nervous system delivery — an area of value to the whole field given that delivery, not editing, is the limiting step for brain indications.
Sources
- New England Journal of Medicine (Tebas et al.) · 2014
Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV ↗