Basic Information

Gene symbol MERTK Synonyms MER, RP38, Tyro12, c-Eyk, c-mer Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas | PertOrg
Description MER proto-oncogene, tyrosine kinase

GTO ID GTC0129
Trial ID NCT01482195
Disease Retinal Disease
Altered gene MERTK
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment rAAV2-VMD2-hMERTK
PhasePhase1
Recruitment statusCompleted
TitlePhase I Trial of Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus (rAAV2-VMD2-hMERTK) Gene Vector to Patients With Retinal Disease Due to MERTK Mutations
Year2011
CountrySaudi Arabia
Company sponsorKing Khaled Eye Specialist Hospital
Other ID(s)0916-P
Vector information
Vectoradeno-associated virus
ConstructrAAV2-VMD2-hMERTK
Vector typerecombinant adeno-associated virus serotype 2 (rAAV2) vector
Transgene/Inserted genehuman MERTK gene
Viral genome modificationrep/cap-deleted
Vector production methodThe vector is formulated in BSS and sterile water for injection.

Clinical Result

Cohort 1
Administration route subretinal injection
Pts 6
Age Child, Adult, Older_Adult
Outcome Visual Acuity Measurement: 3 patients improved VA, 3 patients stable VA at 2 years.
Adverse reactions 3/6(Blood and lymphatic system disorders; Eye disorders)
References PMID: 26825853

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