Engineering Genetic Systems for Treating Mitochondrial Diseases

• Published in: Pharmaceutics Vol. 13; no. 6; p. 810
• Main Authors: Jang, Yoon-ha, Ahn, Sae Ryun, Shim, Ji-yeon, Lim, Kwang-il
• Format: Journal Article
• Language: English
• Published: Basel MDPI AG 28.05.2021; MDPI
• Subjects: Adenosine triphosphate; Alzheimer's disease; Ataxia; Binding sites; Deafness; Gene expression; gene therapy; Genomes; heteroplasmy; Mitochondria; mitochondrial disease; Mitochondrial DNA; mitochondrial gene delivery; Mutation; Phosphorylation; Physiology; Proteins; Review; RNA polymerase
• ISSN: 1999-4923; 1999-4923
• DOI: 10.3390/pharmaceutics13060810

Mitochondria are intracellular energy generators involved in various cellular processes. Therefore, mitochondrial dysfunction often leads to multiple serious diseases, including neurodegenerative and cardiovascular diseases. A better understanding of the underlying mitochondrial dysfunctions of the molecular mechanism will provide important hints on how to mitigate the symptoms of mitochondrial diseases and eventually cure them. In this review, we first summarize the key parts of the genetic processes that control the physiology and functions of mitochondria and discuss how alterations of the processes cause mitochondrial diseases. We then list up the relevant core genetic components involved in these processes and explore the mutations of the components that link to the diseases. Lastly, we discuss recent attempts to apply multiple genetic methods to alleviate and further reverse the adverse effects of the core component mutations on the physiology and functions of mitochondria.