The nonsense suppression drug PTC124 restored alpha-I-iduronidase activity and reduces glycosaminoglycan accumulation in MPS IH mice carrying the Idua-W402X mutation

MOLECULAR GENETICS AND METABOLISM

卷 114, 期 2, 页码 S20-S20

ACADEMIC PRESS INC ELSEVIER SCIENCE

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Endocrinology & Metabolism Genetics & Heredity Medicine, Research & Experimental