CRISPR/Cas9 Edited Hematopoietic Stem and Progenitor Cells for Friedreich’s Ataxia

1354 CRISPR/Cas9 Edited Hematopoietic Stem and Progenitor Cells for Friedreich’s Ataxia; Anusha Sivakumar, Rafael Andres Badell-Grau, Rita Wan, Veenita Khare, Stephanie Cherqui. May 01, 2023 Volume 31Issue 4Supplement 1S1-794 26th Annual Meeting of the American Society of Gene & Cell Therapy.

Preliminary data suggest decreased pathological cardiac hypertrophy, improved mitochondrial biogenesis, reduced inflammation, and oxidativedamage in mice receiving gene-edited cells compared to mock. Altogether, the preliminary data from the in vivo studies demonstrate that single infusion of FXN gene corrected HSPCs engraft in the bone marrow niche to become a reservoir of healthy cells that can integrate into the injured organs for local and systemic delivery of frataxin protein.

CRISPR/Cas9 Edited Hematopoietic Stem and Progenitor Cells for Friedreich’s Ataxia