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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 Share Save
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Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders Davidson, Beverly L.; Gao, Guangping; Berry-Kravis, Elizabeth; Bradbury, Allison M.; Bonnemann, Carsten; Buxbaum, Joseph D.; Corcoran, Gavin R.; Gray, Steven J.; Gray-Edwards, Heather; Kleiman, Robin J.; Shaywitz, Adam J.; Wang, Dan; Zoghbi, Huda Y.; Flotte, Terence R.; Tauscher-Wisniewski, Sitra; Tifft, Cynthia J.; Sahin, Mustafa Share Save
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AAV gene therapy for Tay-Sachs disease Flotte, Terence R.; Cataltepe, Oguz; Puri, Ajit; Batista, Ana Rita; Moser, Richard; McKenna-Yasek, Diane; Douthwright, Catherine; Gernoux, Gwladys; Blackwood, Meghan; Mueller, Christian; Tai, Phillip W. L.; Jiang, Xuntian; Bateman, Scot; Spanakis, Spiro G.; Parzych, Julia; Keeler, Allison M.; Abayazeed, Aly; Rohatgi, Saurabh; Gibson, Laura; Finberg, Robert; Barton, Bruce A.; Vardar, Zeynep; Shazeeb, Mohammed Salman; Gounis, Matthew; Tifft, Cynthia J.; Eichler, Florian S.; Brown, Robert H., Jr.; Martin, Douglas R.; Gray-Edwards, Heather L.; Sena-Esteves, Miguel Share Save
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Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice Liu, Pengpeng; Liang, Shun-Qing; Zheng, Chunwei; Mintzer, Esther; Zhao, Yan G.; Ponnienselvan, Karthikeyan; Mir, Aamir; Sontheimer, Erik J.; Gao, Guangping; Flotte, Terence R.; Wolfe, Scot A.; Xue, Wen Share Save
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