Not logged in 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
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
EZH2 inhibition reactivates epigenetically silenced FMR1 and normalizes molecular and electrophysiological abnormalities in fragile X syndrome neurons Fang, Minggang; Deibler, Sara K.; Krishnamurthy, Pranathi Meda; Wang, Feng; Rodriguez, Paola; Banday, Shahid; Virbasius, Ching-Man; Sena-Esteves, Miguel; Watts, Jonathan K.; Green, Michael R. Share Save
Adeno-associated viral gene therapy for sialidosis using small and large animal models Gallagher, Jillian; Gallant, Judith; Xu, Ping; Gately, Rachael; Prestigiacomo, Rachel; McElroy, Abigail; Hall, Erin; Benatti, Hector; Fremuth, Leigh V.; van de Vlekkert, Diantha; Toro, Camilo; Tifft, Cynthia J.; Wolfe, Scott; d'Azzo, Alessandra; Sena-Esteves, Miguel; Gray-Edwards, Heather Share Save
Life-Limiting Peripheral Organ Dysfunction in Feline Sandhoff Disease Emerges after Effective CNS Gene Therapy Johnson, Aime K.; McCurdy, Victoria J.; Gray-Edwards, Heather L.; Maguire, Anne S.; Cochran, J. Nicholas; Gross, Amanda L.; Skinner, Haleigh E.; Randle, Ashley N.; Shirley, Jamie L.; Brunson, Brandon L.; Bradbury, Allison M.; Leroy, Stanley G.; Hwang, Misako; Rockwell, Hannah E.; Cox, Nancy R.; Baker, Henry J.; Seyfried, Thomas N.; Sena-Esteves, Miguel; Martin, Douglas R. Share Save
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From mouse to sheep: Generating a sheep model and developing a gene therapy for sialidosis Gallagher, Jillian; Gallant, Judith; Xu, Ping; Gately, Rachael; Bertrand, Stephanie; McElroy, Abigail; Hall, Erin; Benatti, Hector; Prestigiacomo, Rachel; Luk, Kevin; Fremuth, Leigh E.; van de Vlekkert, Diantha; Hu, Huimin; Toro, Camilo; d'Azzo, Alessandra; Tifft, Cynthia J.; Wolfe, Scot; Rivera, Jaime; Sena-Esteves, Miguel; Gray-Edwards, Heather Share Save
Excessive release of inorganic polyphosphate by ALS/FTD astrocytes causes non-cell-autonomous toxicity to motoneurons Arredondo, Cristian; Cefaliello, Carolina; Dyrda, Agnieszka; Jury, Nur; Martinez, Pablo; Diaz, Ivan; Amaro, Armando; Tran, Helene; Morales, Danna; Pertusa, Maria; Stoica, Lorelei; Fritz, Elsa; Corvalan, Daniela; Abarzua, Sebastian; Mendez-Ruette, Maxs; Fernandez, Paola; Rojas, Fabiola; Kumar, Meenakshi Sundaram; Aguilar, Rodrigo; Almeida, Sandra; Weiss, Alexandra; Bustos, Fernando J.; Gonzalez-Nilo, Fernando; Otero, Carolina; Tevy, Maria Florencia; Bosco, Daryl A.; Saez, Juan C.; Kahne, Thilo; Gao, Fen-Biao; Berry, James D.; Nicholson, Katharine; Sena-Esteves, Miguel; Madrid, Rodolfo; Varela, Diego; Montecino, Martin; Brown, Robert H.; van Zundert, Brigitte 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
Sialidosis: From gene editing to gene therapy Gallagher, Jillian; Gallant, Judith; Xu, Ping; Gately, Rachael; Bertrand, Stephanie; Luk, Kevin; Toro, Camilo; d'Azzo, Alessandra; Wolfe, Scot; Rivera, Jaime; Sena-Esteves, Miguel; Gray-Edwards, Heather Share Save
Mutation in protein disulfide isomerase A3 causes neurodevelopmental defects by disturbing endoplasmic reticulum proteostasis Medinas, Danilo Bilches; Malik, Sajid; Yildiz-Bolukbasi, Esra; Borgonovo, Janina; Saaranen, Mirva J.; Urra, Hery; Pulgar, Eduardo; Afzal, Muhammad; Contreras, Darwin; Wright, Madison T.; Bodaleo, Felipe; Quiroz, Gabriel; Rozas, Pablo; Mumtaz, Sara; Diaz, Rodrigo; Rozas, Carlos; Cabral-Miranda, Felipe; Pina, Ricardo; Valenzuela, Vicente; Uyan, Ozgun; Reardon, Christopher; Woehlbier, Ute; Brown, Robert H.; Sena-Esteves, Miguel; Gonzalez-Billault, Christian; Morales, Bernardo; Plate, Lars; Ruddock, Lloyd W.; Concha, Miguel L.; Hetz, Claudio; Tolun, Aslihan Share Save
Natural history of Tay-Sachs disease in sheep Story, Brett; Taghian, Toloo; Gallagher, Jillian; Koehler, Jey; Taylor, Amanda; Randle, Ashley; Nielsen, Kayly; Gross, Amanda; Maguire, Annie; Carl, Sara; Johnson, Siauna; Fernau, Deborah; Diffie, Elise; Cuddon, Paul; Corado, Carly; Chandra, Sundeep; Sena-Esteves, Miguel; Kolodny, Edwin; Jiang, Xuntian; Martin, Douglas; Gray-Edwards, Heather Share Save
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
Upregulating β-hexosaminidase activity in rodents prevents α-synuclein lipid associations and protects dopaminergic neurons from α-synuclein-mediated neurotoxicity Brekk, Oeystein R.; Korecka, Joanna A.; Crapart, Cecile C.; Huebecker, Mylene; MacBain, Zachary K.; Rosenthal, Sara Ann; Sena-Esteves, Miguel; Priestman, David A.; Platt, Frances M.; Isacson, Ole; Hallett, Penelope J. 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
A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system Alterman, Julia F.; Godinho, Bruno M. D. C.; Hassler, Matthew R.; Ferguson, Chantal M.; Echeverria, Dimas; Sapp, Ellen; Haraszti, Reka A.; Coles, Andrew H.; Conroy, Faith; Miller, Rachael; Roux, Loic; Yan, Paul; Knox, Emily G.; Turanov, Anton A.; King, Robert M.; Gernoux, Gwladys; Mueller, Christian; Gray-Edwards, Heather L.; Moser, Richard P.; Bishop, Nina C.; Jaber, Samer M.; Gounis, Matthew J.; Sena-Esteves, Miguel; Pai, Athma A.; DiFiglia, Marian; Aronin, Neil; Khvorova, Anastasia Share Save
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