| 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 | GTC3949 |
| Trial ID | NL9321 |
| Disease | Retinal Degeneration |
| Altered gene | RPE65 |
| Therapeutic/Target gene | Therapeutic gene |
| Therapy | Gene transfer |
| Treatment | voretigene neparvovec|Luxturna |
| Recruitment status | Pending |
| Title | Prospective study on safety and efficacy of gene therapy with voretigene neparvovec (Luxturna) in patients with RPE65-associated inherited retinal degenerations |
| Year | 2021 |
| Country | Dutch |
| Company sponsor | The Rotterdam Eye Hospital |
| Other ID(s) | NL76234.000.21 |
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| Cohort 1 | |||||
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