Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
この試験で検証していること
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 ↗
試験結果が意味すること・意味しないこと
初期段階の結果は少人数の参加者と短い追跡期間から得られたものです。参加者が増え時間が経つにつれて変わる可能性があり、募集中・実施中・完了済みの試験であっても、治療効果が確立されたわけではありません。登録基準は厳格で、適格性の判断は試験実施施設が行うものであり、ウェブサイトが行うものではありません。