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Identification of a novel compound heterozygous IDUA mutation underlies Mucopolysaccharidoses type I in a Chinese pedigree

BACKGROUND: Mucopolysaccharidosis type I (MPS I) is a rare autosomal storage disorder resulting from the defective alpha‐L‐iduronidase (encoded by IDUA) enzyme activity and accumulation of glycosaminoglycans (GAGs) in lysosomes. So far, more than 100 IDUA causative mutations have been identified lea...

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Bibliografske podrobnosti
izdano v:Mol Genet Genomic Med
Main Authors: Zhou, Yong‐An, Li, Ping, Zhang, Yanping, Xiong, Qiuhong, Li, Chao, Zhao, Zhonghua, Wang, Yuxian, Xiao, Han
Format: Artigo
Jezik:Inglês
Izdano: John Wiley and Sons Inc. 2019
Teme:
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC6978265/
https://ncbi.nlm.nih.gov/pubmed/31758674
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/mgg3.1058
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