CRISPR-Cas9 gene-edited HSPCs for Friedreich’s ataxia

admin_vjr_user — Fri, 13 Aug 2021 12:05:06 +0000

Stephanie Cherqui, PhD, University of California, San Diego, La Jolla, CA, describes a CRISPR-Cas9 gene-edited hematopoietic stem and progenitor cell (HSPC)-based therapy for Friedreich’s ataxia (FRDA), a rare mitochondrial neurodegenerative disorder. The transplantation of wild-type HSPCs into an FRDA mouse model led to the successful prevention of neurodegeneration in the dorsal root ganglia. Given that FRDA is predominantly caused by a GAA repeat expansion mutation within the frataxin gene (FXN), CRISPR-Cas 9 technology was used to excise the GAA expansion mutation from patient-derived HSPCs, resulting in the restoration of frataxin expression. Future research aims to evaluate the clinical potential of this autologous therapy for FRDA. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.

Friedreich’s Ataxia – VJRegenMed

CRISPR-Cas9 gene-edited HSPCs for Friedreich’s ataxia