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Research

Grant Award Recipients

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iPSC-derived motor neurons cells to determine muscledependent toxicity

Recent findings from our lab predict that muscle-directed therapies hold great promise as definitive treatments for SBMA motor neuron degeneration. Therefore, our goal in this proposal will be to use our well established human SBMA induced pluripotent stem cell (iPSC) model system to generate a human-based muscle toxicity model and recapitulate the noncell autonomous pathogenesis in SBMA. We will co-culture the iPSC-derived muscles with our iPSC-derived motor neurons cells to determine muscledependent toxicity by assaying motor neuron survival and mitochondrial membrane potential in the respective co-culture conditions. Our iPSC model for SBMA will allow us to use a stem cell approach to evaluate muscle dependent non-cell autonomous motor neuron degeneration for the first
time.

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