Open AccessMitochondrial Diseases: Hope for the Future
Author(s) -
Oliver M. Russell,
Gráinne S. Gorman,
Robert N. Lightowlers,
Douglass M. Turnbull
Publication year - 2020
Publication title -
cell
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 26.304
H-Index - 776
eISSN - 1097-4172
pISSN - 0092-8674
DOI - 10.1016/j.cell.2020.02.051
Subject(s) - biology , mitochondrial disease , mitochondrial dna , mitochondrion , mitochondrial fusion , disease , gene , genetics , nuclear gene , genome , human mitochondrial genetics , mutation , germline , dnaja3 , computational biology , bioinformatics , pathology , medicine
Mitochondrial diseases are clinically heterogeneous disorders caused by a wide spectrum of mutations in genes encoded by either the nuclear or the mitochondrial genome. Treatments for mitochondrial diseases are currently focused on symptomatic management rather than improving the biochemical defect caused by a particular mutation. This review focuses on the latest advances in the development of treatments for mitochondrial disease, both small molecules and gene therapies, as well as methods to prevent transmission of mitochondrial disease through the germline.
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