
The Immune Problem at the Heart of AAV Gene Therapy – and Why It Matters More for the Healthy Than for the Sick
Abstract Few developments in the history of medicine have been as quietly revolutionary as adeno-associated virus (AAV) gene therapy. The pace of progress over the past decade has been, by any measure, extraordinary. AAV vectors have enabled some of the most important gene-therapy advances of the last decade: treatments for inherited retinal disease, spinal muscular atrophy, hemophilia, Duchenne muscular dystrophy, aromatic L-amino acid decarboxylase deficiency, and other serious genetic disorders