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AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 Trial Lewis, Connor J.; D'souza, Precilla; Johnston, Jean M.; Acosta, Maria T.; Farmer, Cristan; Baker, Eva H.; Crowell, Anna; Mojica, Yoliann; Ashraf, Sumaiya; Joseph, Lisa; Vezina, Gilbert; Quezado, Zenaide; Yousef, Muhammad H.; Vardar, Zeynep; Shazeeb, Mohammed Salman; Corti, Manuela; Blackwood, Meghan; Coleman, Kirsten; Batista, Rita; Thurm, Audrey; De Boever, Erika; Gahl, William A.; Byrne, Barry J.; Flotte, Terence R.; Jiang, Xuntian; Gross, Amanda L.; Keeler, Allison M.; Gray-Edwards, Heather; Martin, Douglas R.; Sena-Esteves, Miguel; Tifft, Cynthia J. Share Save
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
CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro Meijboom, Katharina E.; Abdallah, Abbas; Fordham, Nicholas P.; Nagase, Hiroko; Rodriguez, Tomas; Kraus, Carolyn; Gendron, Tania F.; Krishnan, Gopinath; Esanov, Rustam; Andrade, Nadja S.; Rybin, Matthew J.; Ramic, Melina; Stephens, Zachary D.; Edraki, Alireza; Blackwood, Meghan T.; Kahriman, Aydan; Henninger, Nils; Kocher, Jean-Pierre A.; Benatar, Michael; Brodsky, Michael H.; Petrucelli, Leonard; Gao, Fen-Biao; Sontheimer, Erik J.; Brown, Robert H.; Zeier, Zane; Mueller, Christian Share Save
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
SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS Mueller, Christian; Berry, James D.; McKenna-Yasek, Diane M.; Gernoux, Gwladys; Owegi, Margaret A.; Pothier, Lindsay M.; Douthwright, Catherine L.; Gelevski, Dario; Luppino, Sarah D.; Blackwood, Meghan; Wightman, Nicholas S.; Oakley, Derek H.; Frosch, Matthew P.; Flotte, Terrence R.; Cudkowicz, Merit E.; Brown, Robert H., Jr. Share Save
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