La guida definitiva all'editing genico.
★
Menu
Home Scopri Notizie Chiedi all'Atlas
Esplora Tecnologie Malattie Trattamenti Sperimentazioni cliniche Aziende Scienziati Geni Ricerca Istituzioni
Oltre la medicina Agricoltura Etica Investimenti Mappa del mondo
Scopri e strumenti Inizia da qui Glossario A–Z Confronta le tecnologie Cronologia Elenchi e classifiche Agenti IA ★ Salvato API
Informazioni Chi siamo Metodologia Fonti dei dati Politica editoriale Contatti Note legali

🧭 Vista guidata
Sei nuovo alla genetica? Spieghiamo ogni termine mentre navighi, in parole semplici. Le stesse pagine, con il supporto già incluso.

⚡ Parere degli esperti
Conosci già la biologia. Solo i contenuti — chiari e compatti, senza spiegazioni aggiuntive. Questa è la visualizzazione predefinita.

Lingua dell'interfaccia
Modalità chiara

Treatment · Inherited blindness

EDIT-101 (Brilliance)

The first CRISPR therapy injected directly into a human organ — scientifically encouraging, and discontinued because the eligible population was too small.

Discontinued in vivoeyediscontinuedCEP290
Clinical research Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.

Spiegazione semplice

EDIT-101 was injected under the retina to cut out a faulty piece of a gene causing a severe inherited blindness. Some patients saw meaningfully better. The programme was then stopped — not because it failed, but because so few people in the world have that exact mutation that developing it further did not make commercial sense. It is the clearest example in the field of good science ending for economic reasons.

Approfondisci

EDIT-101 delivered SpCas9 and dual guides by subretinal AAV5 injection to excise the intronic CEP290 c.2991+1655A>G variant causing Leber congenital amaurosis type 10. The Phase 1/2 BRILLIANCE trial reported clinically meaningful vision improvement in a subset of participants with an acceptable safety profile, published in the New England Journal of Medicine. Editas discontinued development, citing the small eligible population, and subsequently reset its pipeline around in vivo cardiovascular targets.

Sources

Written by The CRISPR Atlas editorial team Last updated Aug 25, 2026

Connected in the Atlas

Every entry on this site is linked to the others it relates to. These connections are part of the record, not a search result.

Technologies

CRISPR-Cas9

Diseases

Inherited Blindness

Clinical Trials

Single Ascending Dose Study of EDIT-101 in Participants With Leber Congenital Amaurosis Type 10

Genes

CEP290

Companies

Editas Medicine
Solo informazioni educative Questa pagina è una risorsa di riferimento, non una consulenza medica. Lo stato della ricerca e delle autorizzazioni regolatorie cambia; controlla la data dell'ultimo aggiornamento in alto e verifica le informazioni importanti nelle fonti primarie elencate.