Triamterene Functions as an Effective Nonsense Suppression Agent for MPS I-H (Hurler Syndrome)
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DOI: 10.3390/ijms24054521
Abstract: 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… read more here.
Keywords: suppression agent; nonsense suppression; triamterene functions; functions effective ... See more keywords