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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Detalles Bibliográficos
Autores principales: Siddiqui, Amna, Dundar, Halil, Sharma, Jyoti, Kaczmarczyk, Aneta, Echols, Josh, Dai, Yanying, Sun, Chuanxi Richard, Du, Ming, Liu, Zhong, Zhao, Rui, Wood, Tim, Sanders, Shalisa, Rasmussen, Lynn, Bostwick, James Robert, Augelli-Szafran, Corinne, Suto, Mark, Rowe, Steven M., Bedwell, David M., Keeling, Kim M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10003437/
https://www.ncbi.nlm.nih.gov/pubmed/36901952
http://dx.doi.org/10.3390/ijms24054521

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