Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy.

Angela Lek <a class=“affiliation-link” title=“From the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., …