N Engl J Med 2017 11;377(18):1713-1722
From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).