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Alexis N. Corl

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#gene editing Open access Oct 2026

Edited HSPCs rescue frataxin deficiency and inflammation-driven multisystemic pathology in Friedreich's ataxia mice.

Friedreich's ataxia (FRDA) is a multisystemic neurodegenerative disorder caused by GAA repeat expansion in intron 1 of frataxin (FXN), resulting in FXN deficiency and mitochondrial dysfunction. Current therapies do not correct the underlying genetic defect. Here, we demonstrate that ex vivo CRISPR/Cas9 excision of the...

Anusha Sivakumar, Alexis N. Corl, Keneth Jordan Muñoz et al. · 1 citation

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