Considerations for Systemic Use of Gene Therapy

Affiliations

• ¹ Powell Gene Therapy Center and Child Health Research Institute, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA. Electronic address: barry.byrne@ufl.edu.
• ² Powell Gene Therapy Center and Child Health Research Institute, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA.
• ³ Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health, London, UK; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK.