Therapeutics Development for Alagille Syndrome

by Phillip Sanchez, Atena Farkhondeh, Ivan Pavlinov, Karsten Baumgaertel, Steven Rodems, Wei Zheng
Published 2021-08-01

Generation of two gene corrected human isogenic iPSC lines (NCATS-CL6104 and NCATS-CL6105) from a patient line (NCATS-CL6103) carrying a homozygous p.R401X mutation in the NGLY1 ge...

by Ivan Pavlinov, Atena Farkhondeh, Shu Yang, Miao Xu, Yu-Shan Cheng, Jeanette Beers, Jizhong Zou, Chengyu Liu, Matthew Might, Steven Rodems, Karsten Baumgärtel, Wei Zheng
Published 2021-10-01