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 first time CRISPR was injected directly into a human organ — under the retina, to remove a faulty piece of a gene causing severe inherited blindness. Some participants saw meaningfully better. The programme was later discontinued because too few people in the world have that exact mutation.
An open-label, single ascending dose Phase 1/2 study of EDIT-101, delivered by subretinal AAV5 injection, in participants with CEP290-associated Leber congenital amaurosis type 10. Published results reported clinically meaningful improvement in visual function in a subset of participants with an acceptable safety profile. Editas subsequently discontinued development for population-size reasons.
Sources
- New England Journal of Medicine · 2024
Gene editing for CEP290-associated retinal degeneration ↗
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.