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
Summary
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. Copyright © 2021 The Author(s). Published by Elsevier B.V. All rights reserved.
| Authors | Son D, Zheng J, Kim IY, You S |
|---|---|
| Journal | Stem cell research |
| Publication Date | 2021 Jul;54:102392 |
| PubMed | 34091428 |
| DOI | 10.1016/j.scr.2021.102392 |