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Scientists Receive $4.8M to Pursue Gene Therapy for ‘Incurable’ Disease

On November 29, 2022, the California Institute for Regenerative Medicine (CIRM) awarded Dr Stephanie Cherqui and colleagues at the University of California San Diego a grant of $4.8 million to move a gene therapy approach aiming for one-time, lifelong treatment of Friedreich’s ataxia closer to clinical trials. In 2017, Dr Cherqui published findings that showed a single infusion of wildtype hematopoietic stem and progenitor cells (HSPCs) into a mouse model of FA measurably halted cellular damage, suggesting a therapeutic approach to FA. In 2020, Cherqui and colleagues published findings that specifically described how CRISPR-Cas9 gene editing of hematopoietic stem cells from patients with FA could work. This study was funded in part by FARA.

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