RNAseq analysis for the diagnosis of muscular dystrophy

نویسندگان

  • Hernan Gonorazky
  • Minggao Liang
  • Beryl Cummings
  • Monkol Lek
  • Johann Micallef
  • Cynthia Hawkins
  • Raveen Basran
  • Ronald Cohn
  • Michael D. Wilson
  • Daniel MacArthur
  • Christian R. Marshall
  • Peter N. Ray
  • James J. Dowling
چکیده

The precise genetic cause remains elusive in nearly 50% of patients with presumed neurogenetic disease, representing a significant barrier for clinical care. This is despite significant advances in clinical genetic diagnostics, including the application of whole-exome sequencing and next-generation sequencing-based gene panels. In this study, we identify a deep intronic mutation in the DMD gene in a patient with muscular dystrophy using both conventional and RNAseq-based transcriptome analyses. The implications of our data are that noncoding mutations likely comprise an important source of unresolved genetic disease and that RNAseq is a powerful platform for detecting such mutations.

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عنوان ژورنال:

دوره 3  شماره 

صفحات  -

تاریخ انتشار 2016