Engineering Genetic Systems for Treating Mitochondrial Diseases
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초록

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.

키워드

mitochondrial diseasegene therapymitochondrial DNAheteroplasmymitochondrial gene deliveryHEREDITARY OPTIC NEUROPATHYLIPOSOME-BASED CARRIERDNA-POLYMERASE-GAMMARIBOSOMAL-RNA IMPORTD-LOOPOXIDATIVE-PHOSPHORYLATIONDQASOME/DNA COMPLEXESBIOACTIVE MOLECULESCOPY NUMBERPLASMID DNA
제목
Engineering Genetic Systems for Treating Mitochondrial Diseases
저자
Jang, Yoon-haAhn, Sae RyunShim, Ji-yeonLim, Kwang-il
DOI
10.3390/pharmaceutics13060810
발행일
2021-06
유형
Review
저널명
Pharmaceutics
13
6
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1 ~ 20