Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
O que este estudo está testando
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 ↗
O que um resultado de estudo significa — e o que não significa
Resultados de fases iniciais vêm de um número pequeno de participantes e acompanhamento de curto prazo. Eles podem mudar com mais pessoas e mais tempo, e um ensaio que está recrutando, ativo ou até concluído ainda não estabeleceu que um tratamento funciona. Os critérios de inclusão são rigorosos; a elegibilidade é decidida pelo centro do ensaio, nunca por um site.