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Paper Details

Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy.
11
2023
DMD, Duchenne's Muscular Dystrophy, Duchenne's muscular dystrophy, Patient, dCas9, patient
Author NameAffiliation
Monkol LekYale School of Medicine, T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., University of Massachusetts Chan Medical School, Boston Children's Hospital, and Harvard Medical School (J.P., University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu
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