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:


  1. Diagnosis of retinitis pigmentosa caused by MERTK mutations
  2. 14 to 70 years of age
  3. Visual acuity of 20/100 or less in worse eye


Clinical Contacts:

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

Additional Information:

MERTK gene therapy clinical trial listing on