在高剂量rAAV9基因治疗后死亡,患者患有杜氏肌肉发育不良症

Angela Lek¹, Brenda Wong¹, Allison Keeler¹+22

• ¹From 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., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).