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Gene Augmentation and Readthrough Rescue Channelopathy in an iPSC-RPE Model of Congenital Blindness |
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| Authors: | Pawan K. Shahi Dalton Hermans Divya Sinha Simran Brar Hannah Moulton Sabrina Stulo Katarzyna D. Borys Elizabeth Capowski De-Ann M. Pillers David M. Gamm Bikash R. Pattnaik |
| Affiliation: | 1. Division of Neonatology, Department of Pediatrics, University of Wisconsin-Madison, Madison, WI 53706, USA;2. McPherson Eye Research, University of Wisconsin-Madison, Madison, WI 53705, USA;3. Waisman Center, University of Wisconsin-Madison, Madison, WI 53705, USA;4. Medical Genetics, University of Wisconsin-Madison, Madison, WI 53706, USA;5. Department of Ophthalmology and Visual Sciences, University of Wisconsin-Madison, Madison, WI 53705, USA |
| Abstract: | |
| Keywords: | ion-channels induced pluripotent stem cells (iPSC) Leber congenital amaurosis (LCA) KCNJ13 Kir7.1 retinal pigment epithelium (RPE) gene-therapy translational readthrough loss-of-function blindness genetic disorders |
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