Gene symbol | RPE65 | Synonyms | BCO3, LCA2, RP20, mRPE65, p63, rd12, sRPE65 | Type of gene | protein-coding |
Chromosome | 1 | Map location | 1p31.3 | dbXrefs | |
Description | retinoid isomerohydrolase RPE65 |
GTO ID | GTC1930 |
Trial ID | NCT03597399 |
Disease | Retinal Dystrophy |
Altered gene | RPE65 |
Therapeutic/Target gene | Therapeutic gene |
Therapy | Gene transfer |
Treatment | voretigene neparvovec|Luxturna |
Recruitment status | Active, Not Recruiting |
Title | A Post-Authorization, Multicenter, Longitudinal, Observational Safety Registry Study for Patients Treated With Voretigene Neparvovec in US |
Year | 2018 |
Country | United States |
Company sponsor | Spark Therapeutics, Inc. |
Other ID(s) | SPKRPE-PASS |
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