Unknown

Dataset Information

0

Toward a therapy for mitochondrial disease.


ABSTRACT: Mitochondrial disorders are a group of genetic diseases affecting the energy-converting process of oxidative phosphorylation. The extreme variability of symptoms, organ involvement, and clinical course represent a challenge to the development of effective therapeutic interventions. However, new possibilities have recently been emerging from studies in model organisms and awaiting verification in humans. I will discuss here the most promising experimental approaches and the challenges we face to translate them into the clinics. The current clinical trials will also be briefly reviewed.

SUBMITTER: Viscomi C 

PROVIDER: S-EPMC5095900 | biostudies-literature | 2016 Oct

REPOSITORIES: biostudies-literature

altmetric image

Publications

Toward a therapy for mitochondrial disease.

Viscomi Carlo C  

Biochemical Society transactions 20161001 5


Mitochondrial disorders are a group of genetic diseases affecting the energy-converting process of oxidative phosphorylation. The extreme variability of symptoms, organ involvement, and clinical course represent a challenge to the development of effective therapeutic interventions. However, new possibilities have recently been emerging from studies in model organisms and awaiting verification in humans. I will discuss here the most promising experimental approaches and the challenges we face to  ...[more]

Similar Datasets

| S-EPMC3808129 | biostudies-literature
2010-06-24 | E-GEOD-18677 | biostudies-arrayexpress
| S-EPMC5220060 | biostudies-literature
| S-EPMC4860742 | biostudies-literature
| S-EPMC3262379 | biostudies-other
| S-EPMC4096714 | biostudies-literature
| S-EPMC2829286 | biostudies-literature
| S-EPMC6195631 | biostudies-literature
| S-EPMC11372595 | biostudies-literature
2019-11-16 | GSE140483 | GEO