Not logged in Five-year analysis of efficacy and safety ofa bidirectional AAV gene therapy in Tay-Sachs sheep Taghian, Toloo; Gallagher, Jillian; Bertrand, Stephanie; Baker, William C.; Mercado, Kalajan Lopez; Benatti, Hector R.; Hall, Erin; Lopez, Yvette; McElroy, Abigail; McCarthy, John T.; Pulaparthi, Sanjana; Fernau, Deborah; Mather, Samuel; Esteves, Sophia; Diffie, Elise; Gross, Amanda; Lahey, Hannah G.; Jiang, Xuntian; Parsley, Elizabeth; Gately, Rachael; Prestigiacomo, Rachel; Johnson, Siauna; Taylor, Amanda; Bierfeldt, Lindsey; Tuominen, Susan; Koehler, Jennifer; Gao, Guangping; Xie, Jun; Su, Qin; King, Robert; Gounis, Matthew J.; Anagnostakou, Vania; Puri, Ajit; Batista, Ana Rita; Sena-Esteves, Miguel; Martin, Douglas R.; Gray-Edwards, Heather Share Save
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AAV9-Mediated Intrastriatal Delivery of GNAO1 Reduces Hyperlocomotion in Gnao1 Heterozygous R209H Mutant MiceS Roy, Alex J.; Leipprandt, Jeffrey R.; Patterson, Joseph R.; Stoll, Anna C.; Kemp, Christopher J.; Oula, Zaipo-Tcheisian D.; Mola, Tyler; Batista, Ana R.; Sortwell, Caryl E.; Sena-Esteves, Miguel; Neubig, Richard R. Share Save
Focused Ultrasound-Mediated Disruption of the Blood-Brain Barrier for AAV9 Delivery in a Mouse Model of Huntington's Disease Owusu-Yaw, Bernie S.; Zhang, Yongzhi; Garrett, Lilyan; Yao, Alvin; Shing, Kai; Batista, Ana Rita; Sena-Esteves, Miguel; Upadhyay, Jaymin; Kegel-Gleason, Kimberly; Todd, Nick 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
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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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Intravenous delivery of adeno-associated viral gene therapy in feline GM1 gangliosidosis Gross, Amanda L.; Gray-Edwards, Heather L.; Bebout, Cassie N.; Ta, Nathan L.; Nielsen, Kayly; Brunson, Brandon L.; Mercado, Kalajan R. Lopez; Osterhoudt, Devin E.; Batista, Ana Rita; Maitland, Stacy; Seyfried, Thomas N.; Sena-Esteves, Miguel; Martin, Douglas R. Share Save
Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice Lahey, Hannah G.; Webber, Chelsea J.; Golebiowski, Diane; Izzo, Cassandra M.; Horn, Erin; Taghian, Toloo; Rodriguez, Paola; Batista, Ana Rita; Ellis, Lauren E.; Hwang, Misako; Martin, Douglas R.; Gray-Edwards, Heather; Sena-Esteves, Miguel Share Save
A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna Taghian, Toloo; Marosfoi, Miklos G.; Puri, Ajit S.; Cataltepe, Oguz. I.; King, Robert M.; Diffie, Elise B.; Maguire, Anne S.; Martin, Douglas R.; Fernau, Deborah; Batista, Ana Rita; Kuchel, Tim; Christou, Chris; Perumal, Raj; Chandra, Sundeep; Gamlin, Paul D.; Bertrand, Stephanie G.; Flotte, Terence R.; McKenna-Yasek, Diane; Tai, Phillip W. L.; Aronin, Neil; Gounis, Matthew J.; Sena-Esteves, Miguel; Gray-Edwards, Heather L. Share Save
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