23 Apr CRISPR Gene Editing May Lead To Cure For Retinitis Pigmentosa
MedicalResearch.com Interview with:
Kang Zhang, M.D., Ph.D.
Professor of Ophthalmology
Chief, Ophthalmic Genetics
Founding Director, Institute for Genomic Medicine
Co-Director, Biomaterials and Tissue Engineering, Institute for Engineering in Medicine
Board Certification in Ophthalmology
Fellowship in Vitreoretinal Disease and Surgery
Guangzhou Women and Children’s Medical Center
Guangzhou Medical University
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: Retinitis pigmentosa is a common blinding condition characterized by mutations in rod photoreceptor specific genes, night blindness and tunnel visual with eventual loss of day vision. Since it can be caused by numerous different mutations in many genes therefore it has been difficult to provide treatment benefits to a majority of patients. Traditional gene therapy has been in a piece-meal fashion, meaning to create a therapy for a particular gene or mutation. In this paper, we describe a universal gene therapy approach using the latest gene editing technology CRISPR/CAS9 to reprogram rod photoreceptors to cone photoreceptors with reversal of RP and restoration of vision.
MedicalResearch.com: What should readers take away from your report?
Response: Because this simple and straight forward treatment strategy can apply to a majority of Retinitis pigmentosa patients in a very cost-effective manner (as it will only require one gene therapy vector to treat all diseases), and relatively good safety profile of adeno-associated virus vector, we expect a human clinical trial to start soon.
MedicalResearch.com: What recommendations do you have for future research as a result of this study?
Response: Our principle of reprogramming a gene mutation vulnerable to resistant cell can prevent cellular and tissue degeneration can be applied to many other areas in regenerative medicine, such as reprogramming an aggressive cancer cell into a more mature differentiated cells that resists oncogene mutations.
MedicalResearch.com: Thank you for your contribution to the MedicalResearch.com community.
Gene and mutation independent therapy via CRISPR-Cas9 mediated cellular reprogramming in rod photoreceptors
Jie Zhu1,2,*, Chang Ming3,*, Xin Fu1,2,*, Yaou Duan2, Duc Anh Hoang3, Jeffrey Rutgard2, Runze Zhang2, Wenqiu Wang2, Rui Hou4, Daniel Zhang2, Edward Zhang2, Charlotte Zhang2, Xiaoke Hao5, Wenjun Xiong3 and Kang Zhang
Cell Research advance online publication 21 April 2017; doi: 10.1038/cr.2017.57
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Last Updated on April 23, 2017 by Marie Benz MD FAAD