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  • 标题:Diagnosis of a mild peroxisomal phenotype with next-generation sequencing
  • 作者:Meredith J. Ventura ; Dianna Wheaton ; Mingchu Xu
  • 期刊名称:Molecular Genetics and Metabolism Reports
  • 印刷版ISSN:2214-4269
  • 出版年度:2016
  • 卷号:9
  • 页码:75-78
  • DOI:10.1016/j.ymgmr.2016.10.006
  • 出版社:Elsevier B.V.
  • 摘要:

    Peroxisomal biogenesis disorders (PBD) are caused by mutations in PEX genes, and are typically diagnosed with biochemical testing in plasma followed by confirmatory testing. Here we report the unusual diagnostic path of a child homozygous for PEX1 p.G843D. The patient presented with sensorineural hearing loss, pigmentary retinopathy, and normal intellect. After testing for Usher syndrome was negative, he was found to have PBD through a research sequencing panel. When evaluating a patient with hearing loss and pigmentary retinopathy, mild PBD should be on the differential regardless of cognitive function.

  • 关键词:PEX1 p.G843D ; Zellweger syndrome spectrum ; Peroxisomal biogenesis disorders ; Usher syndrome
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