Self-replicative mRNA-mediated generation of induced pluripotent stem cell line from a 1-year-old Leigh syndrome patient with mitochondrial DNA cytochrome b mutation
- Authors
- Son, Daryeon; Zheng, Jie; Kim, In Yong; You, Seungkwon
- Issue Date
- Jul-2021
- Publisher
- ELSEVIER
- Citation
- STEM CELL RESEARCH, v.54
- Indexed
- SCIE
SCOPUS
- Journal Title
- STEM CELL RESEARCH
- Volume
- 54
- URI
- https://scholar.korea.ac.kr/handle/2021.sw.korea/137160
- DOI
- 10.1016/j.scr.2021.102392
- ISSN
- 1873-5061
1876-7753
- Abstract
- Leigh syndrome is a progressive neurodegenerative disease due to defects in the mitochondrial genes, including mitochondrial DNA cytochrome b (MTCYB) mutation, that typically begins in infancy or early childhood. Exercise intolerance and fatigue are common symptoms of mitochondrial disorders. Here, we generated induced pluripotent stem cell (iPSC) line from a 1-year-old patient with Leigh syndrome with MTCYB through temporal expression of exogenes, synthetic self-replicative mRNAs which were regulated by B18R protein. The established iPSCs showed expression of various pluripotency markers, a normal karyotype and differentiation potential to three germ layers in vitro while retaining MTCYB mutation.
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Collections - College of Life Sciences and Biotechnology > ETC > 1. Journal Articles
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