Gene symbol | ABCA4 | Synonyms | ABC10, ABCR, ARMD2, CORD3, FFM, RMP, RP19, STGD, STGD1 | Type of gene | protein-coding |
Chromosome | 1 | Map location | 1p22.1 | dbXrefs | |
Gene perturbation-related omics dataset | PerturbAtlas | PertOrg | ||||
Description | ATP binding cassette subfamily A member 4 |
GTO ID | GTC3794 |
Trial ID | NCT06300476 |
Disease | Stargardt Disease 1 |
Altered gene | ABCA4 |
Therapeutic/Target gene | Therapeutic gene |
Therapy | Gene transfer |
Treatment | JWK006 |
Phase | Phase1|Phase2 |
Recruitment status | Active, Not Recruiting |
Title | Safety and Efficacy of a Single Subretinal Injection of JWK006 Gene Therapy in Subjects With Stargardt Disease(STGD1) |
Year | 2024 |
Country | China |
Company sponsor | West China Hospital |
Other ID(s) | 2023-1511 |
Vector information | |||||||||
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Cohort 1 | |||||||
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