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In vivo survival and differentiation of Friedreich ataxia iPSC-derived sensory neurons transplanted in the adult dorsal root ganglia

Journal Article


Abstract


  • 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.

Publication Date


  • 2021

Citation


  • Viventi, S., Frausin, S., Howden, S. E., Lim, S. Y., Finol-Urdaneta, R. K., McArthur, J. R., . . . Dottori, M. (2021). In vivo survival and differentiation of Friedreich ataxia iPSC-derived sensory neurons transplanted in the adult dorsal root ganglia. Stem Cells Translational Medicine, 10(8), 1157-1169. doi:10.1002/sctm.20-0334

Scopus Eid


  • 2-s2.0-85102637632

Start Page


  • 1157

End Page


  • 1169

Volume


  • 10

Issue


  • 8

Abstract


  • 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.

Publication Date


  • 2021

Citation


  • Viventi, S., Frausin, S., Howden, S. E., Lim, S. Y., Finol-Urdaneta, R. K., McArthur, J. R., . . . Dottori, M. (2021). In vivo survival and differentiation of Friedreich ataxia iPSC-derived sensory neurons transplanted in the adult dorsal root ganglia. Stem Cells Translational Medicine, 10(8), 1157-1169. doi:10.1002/sctm.20-0334

Scopus Eid


  • 2-s2.0-85102637632

Start Page


  • 1157

End Page


  • 1169

Volume


  • 10

Issue


  • 8