期刊
EXPERT OPINION ON BIOLOGICAL THERAPY
卷 22, 期 9, 页码 1209-1219出版社
TAYLOR & FRANCIS LTD
DOI: 10.1080/14712598.2022.2070427
关键词
Mitochondria; drug delivery system; gene therapy; RNA delivery therapy; gene editing; MITO-Porter
资金
- Ministry of Education, Culture, Sports, Science and Technology [20K21872, 20H04523]
- Fusion Oriented Research for disruptive Science and Technology (FOREST) Program from the Japan Science and Technology Agency (JST) [JPMJFR203X]
- Special Education and Research Expenses of the MEXT
- Japanese Government (MEXT)
- Grants-in-Aid for Scientific Research [20H04523, 20K21872] Funding Source: KAKEN
This review summarizes the current state of research on RNA-based therapeutics targeted to mitochondria, emphasizing on mitochondrial RNA delivery therapies and therapies involving the use of mitochondrial genome editing devices. Methods for delivering nucleic acids to mitochondria are needed in order to realize such innovative therapies.
Introduction After the emergence of lipid nanoparticles (LNP) containing therapeutic mRNA as vaccines for use against the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), the clinical usefulness of nucleic acid-encapsulated LNPs is now a fact. In addition to the nucleus and cytoplasm, mitochondria, which have their own genome, are a site where nucleic acids function in the cell. Gene therapies targeting mitochondria are expected to pave the way for the next generation of therapies. Areas covered Methods for delivering nucleic acids to mitochondria are needed in order to realize such innovative therapies. However, only a few reports on delivery systems targeting mitochondria have appeared. In this review, we summarize the current state of research on RNA-based therapeutics targeted to mitochondria, with emphasis on mitochondrial RNA delivery therapies and on therapies that involve the use of mitochondrial genome editing devices. Expert opinion We hope that this review article will focus our attention to this area of research, stimulate more interest in this field of research, and lead to the development of mitochondria-targeted nucleic acid medicine. It has the potential to become a major weapon against urgent and unknown diseases, including SARS-CoV-2 infections.
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