Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia
确定治疗弗里德赖希共济失调心脏表现的安全有效静脉注射AAVrh.10hFXN剂量
期刊:Human Gene Therapy
影响因子:4
doi:10.1089/hum.2023.020
Munoz-Zuluaga, Carlos; Gertz, Monica; Yost-Bido, Melissa; Greco, Alessandria; Gorman, Nicholas; Chen, Alvin; Kooner, Vikrum; Rosenberg, Jonathan B; De, Bishnu P; Kaminsky, Stephen M; Borczuk, Alain; Ricart Arbona, Rodolfo J; Martin, Heather R; Monette, Sebastien; Khanna, Richie; Barth, Jay A; Crystal, Ronald G; Sondhi, Dolan