Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
Cosa sta testando questo trial
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 ↗
Cosa significa (e cosa non significa) il risultato di un trial
I risultati delle fasi iniziali provengono da un numero ridotto di partecipanti e da un follow-up breve. Possono cambiare con più persone e più tempo, e una sperimentazione in fase di arruolamento, attiva o anche completata non ha dimostrato che un trattamento funzioni. I criteri di arruolamento sono rigidi; l'idoneità è stabilita dal centro che conduce la sperimentazione, mai da un sito web.