1. Department of Preclinical Safety/Translational Medicine, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA.
2. Department of Discovery and Investigative Pathology, Novartis Institutes for BioMedical Research, Cambridge, Massachusetts, USA.
3. Department of PK Sciences—Gene Therapy TA, Novartis Institutes for BioMedical Research, Cambridge, Massachusetts, USA.
4. Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA.
5. Novartis Institutes for BioMedical Research, Cambridge, Massachusetts, USA.
6. Novartis Gene Therapies, Inc., Bannockburn, Illinois, USA.
7. Department of Pediatrics, Washington University School of Medicine and St Louis Children's Hospital, St Louis, Missouri, USA.
8. Department of Preclinical Safety, Novartis Pharma AG, Basel, Switzerland.