Pathogenic mtDNA mutations causing mitochondrial myopathy: The need for muscle biopsy

نویسندگان

  • Steven A. Hardy
  • Emma L. Blakely
  • Andrew I. Purvis
  • Mariana C. Rocha
  • Syeda Ahmed
  • Gavin Falkous
  • Joanna Poulton
  • Michael R. Rose
  • Olivia O'Mahony
  • Niamh Bermingham
  • Charlotte F. Dougan
  • Yi Shiau Ng
  • Rita Horvath
  • Doug M. Turnbull
  • Grainne S. Gorman
  • Robert W. Taylor
چکیده

Pathogenic mitochondrial tRNA (mt-tRNA) gene mutations represent a prominent cause of primary mitochondrial DNA (mtDNA)-related disease despite accounting for only 5%-10% of the mitochondrial genome.(1,2) Although some common mt-tRNA mutations, such as the m.3243A>G mutation, exist, the majority are rare and have been reported in only a small number of cases.(3) The MT-TP gene, encoding mt-tRNA(Pro), is one of the less polymorphic mt-tRNA genes, and only 5 MT-TP mutations have been reported as a cause of mitochondrial muscle disease to date (table e-1 at Neurology.org/ng, P6-10). We report 5 patients with myopathic phenotypes, each harboring different pathogenic mutations in the MT-TP gene, highlighting the importance of MT-TP mutations as a cause of mitochondrial muscle disease and the requirement to study clinically relevant tissue.

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عنوان ژورنال:

دوره 2  شماره 

صفحات  -

تاریخ انتشار 2016