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

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

Mucopolysaccharidosis I-Hurler (MPS I-H) is caused by the loss of α-L-iduronidase, a lysosomal enzyme that degrades glycosaminoglycans. Current therapies cannot treat many MPS I-H manifestations. In this study, triamterene, an FDA-approved, antihypertensive diuretic, was found to suppress translatio...

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Bibliographic Details
Main Authors: 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
Format: Article
Language:English
Published: MDPI AG 2023-02-01
Series:International Journal of Molecular Sciences
Subjects:
triamterene
MPS I-H
nonsense suppression
readthrough
<i>IDUA</i>-W402X
Online Access:https://www.mdpi.com/1422-0067/24/5/4521

Internet

https://www.mdpi.com/1422-0067/24/5/4521

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