Safety of Ascending Doses of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich’s Ataxia

1656 Safety of Ascending Doses of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich’s Ataxia; Jonathan B. Rosenberg, Alessandria Greco, Carlos Munoz Zuluaga, Monica La Russa Gertz, Melissa Yost-Bido, Nicholas C. Gorman, Alvin Chen, Vikrum Kooner, Bishnu P. De, Stephen M. Kaminsky, Rodolfo J. Ricart Arbona, Heather R. Martin, Sebastien Monette, Richie Khanna, Ronald G. Crystal, Dolan Sondhi. 

 On average, the increase in cardiac FXN expression after AAVrh.10hFXN administration with 5.7x1011 and 1.8x1012 gc/kg doses was 6.5 and 37%, respectively, over the PBS-treated controls. Together, these data identify the safe doses of AAVrh.10hFXN relevant for the treatment of the cardiac manifestations of FA.

Safety of Ascending Doses of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich’s Ataxia