Dr. Françoise Piguet : Gene therapy of lysosomal storage disorders and new AAV vectors and tools to optimize BBB targeting.
Pr. Stephanie Cherqui : From Cystinosis to Alzheimer’s Disease: Lessons Learned Treating a Rare Metabolic Disease Using Hematopoietic Stem Cell Gene Therapy.
Dr Olivier Biondi : Precision exercise in neuromuscular disorders : disease-specific adaptations on mouse models
