Waisman Center
Advancing knowledge of human development, developmental disabilities, and neurodegenerative diseases.

Gene Augmentation and Readthrough Rescue Channelopathy in an iPSC-RPE Model of Congenital Blindness

Gamm, MD, PhD, D. Gene Augmentation and Readthrough Rescue Channelopathy in an IPSC-RPE Model of Congenital Blindness.

Shahi PK, Hermans D, Sinha D, Brar S, Moulton H, Stulo S, Borys KD, Capowski E, Pillers DM, Gamm DM, Pattnaik BR. (2019). Gene Augmentation and Readthrough Rescue Channelopathy in an iPSC-RPE Model of Congenital Blindness. American Journal of Human Genetics, 104(2):310-318. doi: 10.1016/j.ajhg.2018.12.019.