Gene Therapy for Autosomal Recessive RP Caused by MERTK Mutations
Description: This Phase I clinical trial is evaluating the safety of gene replacement therapy for people with retinitis pigmentosa (RP) caused by mutations in the gene MERTK. Efficacy is being evaluated, as well. Initially, 6 participants (children and young adults) will receive a single subretinal injection of the treatment in one eye. The treatment consists of a corrective MERTK gene, which is delivered to retinal pigment epithelial (RPE) cells by an adeno-associated virus (AAV), a therapeutic man-made virus.
This study is a collaboration of the University of California, San Diego and King Khaled Eye Specialist Hospital.
Trial Status: Recruiting participants.
Articles on MERTK Gene Therapy:
First Gene Therapy Clinical Trial for Recessive RP is Underway
Partial List of Participation Criteria:
- Diagnosis of retinitis pigmentosa caused by MERTK mutations
- 14 to 70 years of age
- Visual acuity of 20/100 or less in worse eye
Fowzan S. Alkuraya, M.D.
Phone: 966 1 442 7875
Deepak Edward, M.D.
Phone: 966 1 482 1234 ext 1362
King Khaled Eye Specialist Hospital
Riyadh, Saudi Arabia 11462
MERTK gene therapy clinical trial listing on ClinicalTrials.gov