Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne’s Muscular Dystrophy
Lek A, Wong B, Keeler A, Blackwood M, Ma K, Huang S, Sylvia K, Batista A, Artinian R, Kokoski D, Parajuli S, Putra J, Carreon C, Lidov H, Woodman K, Pajusalu S, Spinazzola J, Gallagher T, LaRovere J, Balderson D, Black L, Sutton K, Horgan R, Lek M, Flotte T. Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne’s Muscular Dystrophy. New England Journal Of Medicine 2023, 389: 1203-1210. PMID: 37754285, PMCID: PMC11288170, DOI: 10.1056/nejmoa2307798.Peer-Reviewed Original ResearchMeSH KeywordsAdultAntibodiesDystrophinGenetic TherapyHumansMuscular Dystrophy, DuchenneRespiratory Distress SyndromeTransgenesConceptsAcute respiratory distress syndromeDuchenne muscular dystrophyMuscular dystrophyAdvanced Duchenne muscular dystrophySevere diffuse alveolar damageDiffuse alveolar damageRespiratory distress syndromeMild cardiac dysfunctionT cell reactivityInnate immune reactionsVirus serotype 9Gene therapyAlveolar damagePericardial effusionDistress syndromeCardiac dysfunctionPostmortem examinationImmune reactionsRAAV gene therapyBody weightPatientsTherapySerotype 9Recombinant adenoDystrophy