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

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    Version of Record (VoR)
    Author(s)
    Viventi, Serena
    Frausin, Stefano
    Howden, Sara E
    Lim, Shiang Y
    Finol-Urdaneta, Rocio K
    McArthur, Jeffrey R
    Abu-Bonsrah, Kwaku Dad
    Ng, Wayne
    Ivanusic, Jason
    Thompson, Lachlan
    Dottori, Mirella
    Griffith University Author(s)
    Ng, Wayne
    Year published
    2021
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    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 ...
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    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.
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    Journal Title
    STEM CELLS Translational Medicine
    DOI
    https://doi.org/10.1002/sctm.20-0334
    Copyright Statement
    © 2021 The Authors. STEM CELLS TRANSLATIONAL MEDICINE published by Wiley Periodicals LLC on behalf of AlphaMed Press. This is an open access article under the terms of the Creative Commons Attribution License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
    Note
    This publication has been entered in Griffith Research Online as an advanced online version.
    Subject
    Biochemistry and Cell Biology
    Medical Biotechnology
    Clinical Sciences
    dorsal root ganglia
    human pluripotent stem cells
    sensory neurons
    transplantation
    Publication URI
    http://hdl.handle.net/10072/403329
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    • Journal articles

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