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.