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

Lek, Angela, Wong, Brenda, Keeler, Allison, Blackwood, Meghan, Ma, Kaiyue, Huang, Shushu, Sylvia, Katelyn, Batista, A. Rita, Artinian, Rebecca, Kokoski, Danielle, Parajuli, Shestruma, Putra, Juan, Carreon, C. Katte, Lidov, Hart, Woodman, Keryn, Pajusalu, Sander, Spinazzola, Janelle M., Gallagher, Thomas, LaRovere, Joan, Balderson, Diane, Black, Lauren, Sutton, Keith, Horgan, Richard, Lek, Monkol, Flotte, Terence·2023·New England Journal of Medicine
DOI 10.1056/nejmoa2307798

Cited by (320)

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  72. Additional file 1 of Human umbilical cord mesenchymal stem cell-based gene therapy for hemophilia B using scAAV-DJ/8-LP1-hFIXco transduction
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