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  • 标题:A novel mutation in SGSH causing Sanfillipo type 3A Mucopolysaccharidoses in an Indian family
  • 作者:Jyotsna Singh ; P.K. Muhammad ; Sweta Jain
  • 期刊名称:Molecular Genetics and Metabolism Reports
  • 印刷版ISSN:2214-4269
  • 出版年度:2018
  • 卷号:15
  • 页码:124-126
  • DOI:10.1016/j.ymgmr.2018.04.003
  • 出版社:Elsevier B.V.
  • 摘要:

    Mucopolysaccharidoses (MPS) type III also termed as Sanfillipo syndrome, involves defect in enzymes required for degradation of heparan sulphate. We report a clinical case of MPS-III later followed by genetic investigation for MPS-III genes SGSH , NAGLU , HGSNAT and GNS. It allowed us to identify a novel and likely pathogenic variant p. G205R in SGSH . Protein based Inslico prediction and protein modelling suggests aberration of helical structure of SGSH protein and reduced binding affinity for its substrate.

  • 关键词:Mucopolysaccharidoses ; Sanfillipo syndrome ; SGSH
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