Functional characterization of Friedreich ataxia iPS-derived neuronal progenitors and their integration in the adult brain
- Author(s)
- Bird, MJ; Needham, K; Frazier, AE; van Rooijen, J; Leung, J; Hough, S; Denham, M; Thornton, ME; Parish, CL; Nayagam, BA; Pera, M; Thorburn, DR; Thompson, LH; Dottori, M;
- Details
- Publication Year 2014,Volume 9,Issue #7,Page e101718
- Journal Title
- PLoS One
- Publication Type
- Journal Article
- Abstract
- Friedreich ataxia (FRDA) is an autosomal recessive disease characterised by neurodegeneration and cardiomyopathy that is caused by an insufficiency of the mitochondrial protein, frataxin. Our previous studies described the generation of FRDA induced pluripotent stem cell lines (FA3 and FA4 iPS) that retained genetic characteristics of this disease. Here we extend these studies, showing that neural derivatives of FA iPS cells are able to differentiate into functional neurons, which don't show altered susceptibility to cell death, and have normal mitochondrial function. Furthermore, FA iPS-derived neural progenitors are able to differentiate into functional neurons and integrate in the nervous system when transplanted into the cerebellar regions of host adult rodent brain. These are the first studies to describe both in vitro and in vivo characterization of FA iPS-derived neurons and demonstrate their capacity to survive long term. These findings are highly significant for developing FRDA therapies using patient-derived stem cells.
- Publisher
- PLoS one
- Research Division(s)
- Molecular Medicine
- Publisher's Version
- https://doi.org/10.1371/journal.pone.0101718
- Open Access at Publisher's Site
http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0101718- Terms of Use/Rights Notice
- Refer to copyright notice on published article.
Creation Date: 2015-02-26 11:26:53
Last Modified: 2015-03-03 08:17:58