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CUMC and its teaching-hospital affiliate, NewYork-Presbyterian Hospital, are part of an international consortium that was recently formed to bring this PDE6A gene therapy to patients. Pending FDA approval, Phase I/II clinical trials could begin within a year.
The Molecular Medicine paper is titled, "Stem Cell (iPS) Grafts in a Preclinical Model of Retinitis Pigmentosa." The other contributors are Yao Li (CUMC), Yi-Ting Tsai (CUMC), Chun-Wei Hsu (CUMC), Deniz Erol (CUMC), Jin Yang (CUMC), Wen-Hsuan Wu (CUMC), Richard J Davis (CUMC), and Dieter Egli (New York Stem Cell Foundation Laboratory). This study was supported by the NYSTEM co26448, National Institutes of Health (grants 5P30CA013696 and P30EY019007), Research to Prevent Blindness, the Joan and Michael Schneeweiss Stem Cell Fund, and the Foundation Fighting Blindness.
The Human Molecular Genetics paper is titled, "Gene therapy provides long-term visual function in a pre-clinical model of retinitis pigmentosa." The other contributors are Katherine J. Wert (CUMC), Richard J. Davis (CUMC), Javier Sancho-Pelluz (CUMC), and Patsy M. Nishina (Jackson Laboratory, Bar Harbor, Maine). The research was supported by the National Institute of Health (grants 5P30EY019007 and R01EY018213), the National Cancer Institute (grant 5P30CA013696), Research to Prevent Blindness, the Department of Defense (grant TS080017-W81XWH-09-1-0575), the Foundation Fighting Blindness, the Joan and Michael Schneeweiss Stem Cell Fund, the Barbara and Donald Jonas Family Fund, and the Tistou and Charlotte Kerstan Foundation.
The authors of both studies declare no financial or other conflicts of interest.
Columbia University Medical Center
Article adapted by Medical News Today from original press release. Click 'references' tab above for source.
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