論文

査読有り
2016年10月

Mitochondrial diseases

NATURE REVIEWS DISEASE PRIMERS
  • Grainne S. Gorman
  • ,
  • Patrick F. Chinnery
  • ,
  • Salvatore DiMauro
  • ,
  • Michio Hirano
  • ,
  • Yasutoshi Koga
  • ,
  • Robert McFarland
  • ,
  • Anu Suomalainen
  • ,
  • David R. Thorburn
  • ,
  • Massimo Zeviani
  • ,
  • Douglass M. Turnbull

2
開始ページ
1
終了ページ
22
記述言語
英語
掲載種別
研究論文(学術雑誌)
DOI
10.1038/nrdp.2016.80
出版者・発行元
NATURE PUBLISHING GROUP

Mitochondrial diseases are a group of genetic disorders that are characterized by defects in oxidative phosphorylation and caused by mutations in genes in the nuclear DNA (nDNA) and mitochondrial DNA (mtDNA) that encode structural mitochondrial proteins or proteins involved in mitochondrial function. Mitochondrial diseases are the most common group of inherited metabolic disorders and are among the most common forms of inherited neurological disorders. One of the challenges of mitochondrial diseases is the marked clinical variation seen in patients, which can delay diagnosis. However, advances in next-generation sequencing techniques have substantially improved diagnosis, particularly in children. Establishing a genetic diagnosis allows patients with mitochondrial diseases to have reproductive options, but this is more challenging for women with pathogenetic mtDNA mutations that are strictly maternally inherited. Recent advances in in vitro fertilization techniques, including mitochondrial donation, will offer a better reproductive choice for these women in the future. The treatment of patients with mitochondrial diseases remains a challenge, but guidelines are available to manage the complications of disease. Moreover, an increasing number of therapeutic options are being considered, and with the development of large cohorts of patients and biomarkers, several clinical trials are in progress.

リンク情報
DOI
https://doi.org/10.1038/nrdp.2016.80
PubMed
https://www.ncbi.nlm.nih.gov/pubmed/27775730
Web of Science
https://gateway.webofknowledge.com/gateway/Gateway.cgi?GWVersion=2&SrcAuth=JSTA_CEL&SrcApp=J_Gate_JST&DestLinkType=FullRecord&KeyUT=WOS:000397866500001&DestApp=WOS_CPL
ID情報
  • DOI : 10.1038/nrdp.2016.80
  • ISSN : 2056-676X
  • PubMed ID : 27775730
  • Web of Science ID : WOS:000397866500001

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