A regulated NMD mouse model supports NMD inhibition as a viable therapeutic option to treat genetic diseases

by Josh Echols, Amna Siddiqui, Yanying Dai, Viktoria Havasi, Richard Sun, Aneta Kaczmarczyk, Kim M. Keeling
Published 2020-08-01

Triamterene Functions as an Effective Nonsense Suppression Agent for MPS I-H (Hurler Syndrome)

by Amna Siddiqui, Halil Dundar, Jyoti Sharma, Aneta Kaczmarczyk, Josh Echols, Yanying Dai, Chuanxi Richard Sun, Ming Du, Zhong Liu, Rui Zhao, Tim Wood, Shalisa Sanders, Lynn Rasmussen, James Robert Bostwick, Corinne Augelli-Szafran, Mark Suto, Steven M. Rowe, David M. Bedwell, Kim M. Keeling
Published 2023-02-01