Ataluren suppresses a premature termination codon in an MPS I-H mouse.

Dan WangXiaojiao XueGwen GunnMing DuAmna SiddiquiMarla WeetallKim M Keeling

Published in: Journal of molecular medicine (Berlin, Germany) (2022)

Ataluren promotes readthrough of PTCs in a wide variety of contexts. Ataluren reduces glycosaminoglyan storage in MPS I-H cell and mouse models. Ataluren has a bell-shaped dose-response curve and a narrow effective range.

Keyphrases

mouse model
single cell
signaling pathway
cell therapy