Article
Gene therapy in patient-specific stem cell lines and a preclinical model of retinitis pigmentosa with membrane frizzled-related protein defects.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Sept 2014
Li Yao, Wu Wen-Hsuan, Hsu Chun-Wei, Nguyen Huy V, Tsai Yi-Ting, Chan Lawrence, Nagasaki Takayuki, Maumenee Irene H, Yannuzzi Lawrence A, Hoang Quan V, Hua Haiqing, Egli Dieter, Tsang Stephen H
Abstract excerpt
Defects in Membrane Frizzled-related Protein (MFRP) cause autosomal recessive retinitis pigmentosa (RP). MFRP codes for a retinal pigment epithelium (RPE)-specific membrane receptor of unknown function. In patient-specific induced pluripotent stem (iPS)-derived RPE cells, precise levels of MFRP, and its dicistronic partner CTRP5, are critical to the regulation of actin organization. Overexpression of CTRP5 in...
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