Perspectives of the Friedreich ataxia community on gene therapy clinical trials.

Autor: Trantham SJ; Genetics and Genomics Graduate Program, University of Florida, Gainesville, FL 32611, USA.; Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL 32611, USA., Coker MA; Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL 32611, USA., Norman S; Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL 32611, USA., Crowley E; Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL 32611, USA., Berthy J; Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL 32611, USA., Byrne BJ; Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL 32611, USA., Subramony S; Department of Neurology, College of Medicine, University of Florida, Gainesville, FL 32611, USA., Lou X; Department of Biostatistics, College of Public Health and Health Professions, University of Florida, Gainesville, FL 32611, USA., Corti M; Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL 32611, USA.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2023 Dec 18; Vol. 32 (1), pp. 101179. Date of Electronic Publication: 2023 Dec 18 (Print Publication: 2024).