| Gene symbol | CEP290 | Synonyms | 3H11Ag, BBS14, CT87, JBTS5, LCA10, MKS4, NPHP6, POC3, SLSN6, rd16 | Type of gene | protein-coding |
| Chromosome | 12 | Map location | 12q21.32 | dbXrefs | |
| Gene perturbation-related omics dataset | PertOrg | ||||
| Description | centrosomal protein 290 | ||||
| GTO ID | GTC2090 |
| Trial ID | NCT03913143 |
| Disease | Leber Congenital Amaurosis |
| Altered gene | CEP290 |
| Therapeutic/Target gene | Target gene |
| Therapy | ASO |
| Treatment | Sepofarsen|QR-110 |
| Phase | Phase2|Phase3 |
| Recruitment status | Active, Not Recruiting |
| Title | Double-masked, Randomized, Controlled, Multiple-dose Study to Evaluate Efficacy, Safety, Tolerability and Syst. Exposure of QR-110 in Leber's Congenital Amaurosis (LCA) Due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene |
| Year | 2019 |
| Country | Belgium|Brazil|Canada|France|Germany|Italy|Netherlands|United Kingdom|United States |
| Company sponsor | ProQR Therapeutics |
| Other ID(s) | PQ-110-003|2018-003501-25 |
| Vector information | |||
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| Cohort 1 | |||||
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