RP2 Gene Analysis and Therapy

Tae Kwann Park, M.D., Ph.D.

Dear GEGC members,

I am TK Park, a Korean retina specialist, affiliated at Soonchunhyang University Hospital Bucheon South Korea. Recently I came to know Global Eye Genetics Consortium through the introduction by Dr. Takeshi Iwata.

I am writing this short draft to ask you for help. Recently we made a Physician-Patients association called Korean Association for Retinal Degeneration. The purpose to establish KARD was to investigate the genetic mutations causing inherited retinal disease mainly RP and find how to treat these conditions with Gene Therapy, iPSc and ES cells. So, I would like to join GEGC and we have started Next generation sequencing. KARD is composed of about 30 of Physicians and more than 5,000 of IRD patients.

I think KARD could perform NGS rapidly. In addition, for the trial for the treatment, we try to make AAV-based therapeutic gene medicine. Of course, we have to design constructs for therapeutic gene. However, as you know, it takes dozen of years from decision of the first target disease to the clinical trials. So, firstly, I decided to ask to use established AAV vector for IRD to Dr. Paul Sieving, former Director of NEI, NIH and Dr. Zhijian Wu, PI of AAV-RP2.

NEI will collaborate us and we are able to start clinical trials for RP2 patients with NEI vector. I think, for the clinical tirals, phase I/IIa, about 30 RP2 patients will be needed. In Korea, we are not able to find enough number of RP2 patients by this time. So, now, I would like to ask to GEGC members. If all of our asian collegues cooperate, I think we could do the first ocular gene therapy tiral in Asia. And, our asian researchers’ community could contribute to the world. If any person want to contact me, please, E-mail to me. My official email is as belows. I am looking forward to see many messages from researchers in different countries. Finally, I really appreciate Dr. Takeshi Iwata to give me a chance to introduce this plan to all GEGC members.

Sincerely yours.

Tae Kwann Park, MD, PhD
Associate Professor
Department of Ophthalmology, College of Medicine, Soonchunhyang University
Department of Ophthalmology, Soonchunhyang University Hospital Bucheon170, Jomaru-ro, Wonmi-gu, Bucheon-si, Gyeonggi-do, ZIP 14584, South Korea
e-mail; tkpark@schmc.ac.kr
C.P; +82-10-3561-1195
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Fax; +82-32-621-5015