Gene symbol | RPGR | Synonyms | COD1, CORDX1, CRD, PCDX, RP15, RP3, XLRP3, orf15 | Type of gene | protein-coding |
Chromosome | X | Map location | Xp11.4 | dbXrefs | |
Description | retinitis pigmentosa GTPase regulator |
GTO ID | GTC2333 |
Trial ID | NCT04312672 |
Disease | Retinitis Pigmentosa |
Altered gene | RPGR |
Therapeutic/Target gene | Therapeutic gene |
Therapy | Gene transfer |
Treatment | botaretigene sparoparvovec |
Recruitment status | Active, Not Recruiting |
Title | Long Term Follow-Up Study of Participants Following an Open Label, Multi-Centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-Associated Virus Vector (AAV2-RPGR) for Gene Therapy of Adults and Children With X-Linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR) |
Year | 2019 |
Country | United Kingdom|United States |
Company sponsor | MeiraGTx UK II Ltd |
Other ID(s) | MGT010 |
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