Open AccessIn vivo survival and differentiation of Friedreich ataxia iPSC ‐derived sensory neurons transplanted in the adult dorsal root ganglia
Author(s) -
Viventi Serena,
Frausin Stefano,
Howden Sara E.,
Lim Shiang Y.,
FinolUrdaneta Rocio K.,
McArthur Jeffrey R.,
AbuBonsrah Kwaku Dad,
Ng Wayne,
Ivanusic Jason,
Thompson Lachlan,
Dottori Mirella
Publication year - 2021
Publication title -
stem cells translational medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.781
H-Index - 71
eISSN - 2157-6580
pISSN - 2157-6564
DOI - 10.1002/sctm.20-0334
Subject(s) - induced pluripotent stem cell , biology , frataxin , embryonic stem cell , progenitor cell , transplantation , ataxia , neurodegeneration , stem cell , pathology , neuroscience , medicine , microbiology and biotechnology , disease , mitochondrion , genetics , aconitase , gene
Friedreich ataxia (FRDA) is an autosomal recessive disease characterized by degeneration of dorsal root ganglia (DRG) sensory neurons, which is due to low levels of the mitochondrial protein Frataxin. To explore cell replacement therapies as a possible approach to treat FRDA, we examined transplantation of sensory neural progenitors derived from human embryonic stem cells (hESC) and FRDA induced pluripotent stem cells (iPSC) into adult rodent DRG regions. Our data showed survival and differentiation of hESC and FRDA iPSC‐derived progenitors in the DRG 2 and 8 weeks post‐transplantation, respectively. Donor cells expressed neuronal markers, including sensory and glial markers, demonstrating differentiation to these lineages. These results are novel and a highly significant first step in showing the possibility of using stem cells as a cell replacement therapy to treat DRG neurodegeneration in FRDA as well as other peripheral neuropathies.