AI Article Synopsis
- Friedreich's ataxia (FRDA) is a rare neurodegenerative disease linked to a GAA triplet repeat expansion in the FXN gene.
- Researchers created induced pluripotent stem cell (iPSC) lines from skin fibroblasts of three different FRDA patients using safe, non-integrating vectors.
- The generated iPSC lines showed essential characteristics of pluripotency, including normal genetic structure and the ability to differentiate into all three germ layers in a lab test.
Article Abstract
Friedreich's ataxia (FRDA) is a rare neurodegenerative disorder which is caused by triplet repeat expansion (GAA) in the first intron of FXN gene. In this present study, we generated induced pluripotent stem cells (iPSC) lines from fibroblasts of three unrelated FRDA patients using integration-free episomal vectors. All iPSC lines express the pluripotency markers such as OCT4 and SSEA4, display normal karyotypes and can differentiate into all three germ layers via in vivo teratoma formation assay.
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| http://dx.doi.org/10.1016/j.scr.2021.102438 | DOI Listing |
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