Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
Qué está evaluando este ensayo
The trial that started clinical gene editing. Years before CRISPR was a tool, patients with HIV had their own immune cells taken out, had a gene edited with zinc finger nucleases, and had them returned. It showed that a permanent, targeted edit could be made in a patient's cells and given back safely.
A Phase 1 study of autologous CD4 T cells with CCR5 disrupted by zinc finger nucleases in people with HIV. Published in the New England Journal of Medicine, it reported that infusion was safe and that modified cells persisted, establishing the clinical feasibility of therapeutic genome editing.
Sources
- New England Journal of Medicine · 2014
Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV ↗
Qué significa y qué no significa el resultado de un ensayo
Los resultados de fases tempranas provienen de un número reducido de participantes y un seguimiento breve. Pueden cambiar con más personas y más tiempo, y un ensayo en reclutamiento, activo o incluso completado no ha demostrado que un tratamiento funcione. Los criterios de inscripción son estrictos; la elegibilidad la decide el centro del ensayo, nunca un sitio web.