The livers of KO mice treated with AAV-PGK-hFXN displayed Kupffer cell hypertrophy and minimal hepatocyte necrosis. These liver changes are commonly associated with AAV gene therapies, are likely related to gene expression, and are considered non-adverse given the low incidence of observations. Overall, dose-dependent efficacy was observed in AAV-PGK-hFXN-treated KO mice compared with vehicle-treated mice based on survival, cardiac function, cardiac injury biomarkers, and histology. These data support the use AAV-based gene transfer as a promising approach to treat patients with FA cardiomyopathy.
Wednesday, May 17, 2023
Reversal of Cardiac Phenotype in a Mouse Model of Friedreich’s Ataxia Following Administration of AAV Gene Therapy
1184 Reversal of Cardiac Phenotype in a Mouse Model of Friedreich’s Ataxia Following Administration of AAV Gene Therapy; Joshua C. Chang, Whitney Blankenberger, Su Liu, Marie Stark, Jacqueline A. Brassard, Tianbi Zhang, Fria Bolan, Samuel D. Sutton, Mario Guerrero, José A. Corleto, Nakyo Heo, Dwaipayan Sen, Mark A. Champe, Fabrizia Urbinati, Bala Medicherla, Carlos Fonck (Astellas Gene Therapies); May 01, 2023 Volume 31Issue 4Supplement 1S1-794 26th Annual Meeting of the American Society of Gene & Cell Therapy
