Life‐Limiting Peripheral Organ Dysfunction in Feline Sandhoff Disease Emerges after Effective CNS Gene Therapy von 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.

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Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System von McElroy, Abigail, Sena-Esteves, Miguel, Arjomandnejad, Motahareh, Keeler, Allison M., Gray-Edwards, Heather L.

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Natural history study of glycan accumulation in large animal models of GM2 gangliosidoses von Cavender, Catlyn, Mangini, Linley, Van Vleet, Jeremy L, Corado, Carley, McCullagh, Emma, Gray-Edwards, Heather L, Martin, Douglas R, Crawford, Brett E, Lawrence, Roger

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PEA15 loss of function and defective cerebral development in the domestic cat von Graff, Emily C, Cochran, J Nicholas, Kaelin, Christopher B, Day, Kenneth, Gray-Edwards, Heather L, Watanabe, Rie, Koehler, Jey W, Falgoust, Rebecca A, Prokop, Jeremy W, Myers, Richard M, Cox, Nancy R, Barsh, Gregory S, Martin, Douglas R

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Abnormal epiphyseal development in a feline model of Sandhoff disease von McNulty, Margaret A., Prevatt, Patricia B., Nussbaum, Elizabeth R., Randle, Ashley N., Johnson, Aime K., Hudson, Judith A., Gray‐Edwards, Heather L., Sena‐Esteves, Miguel, Martin, Douglas R., Carlson, Cathy S.

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Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation von Corti, Manuela, Byrne, Barry J., Gessler, Dominic J., Thompson, Grace, Norman, Samantha, Lammers, Jenna, Coleman, Kirsten E., Liberati, Cristina, Elder, Melissa E., Escolar, Maria L., Tuna, Ibrahim S., Mesaros, Clementina, Kleiner, Gary I., Barbouth, Deborah S., Gray-Edwards, Heather L., Clement, Nathalie, Cleaver, Brian D., Gao, Guangping

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AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates von Hocquemiller, Michaël, Giersch, Laura, Mei, Xin, Gross, Amanda L., Randle, Ashley N., Gray-Edwards, Heather L., Hudson, Judith A., Todeasa, Sophia, Stoica, Lorelei, Martin, Douglas R., Sena-Esteves, Miguel, Aiach, Karen, Laufer, Ralph

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Adeno-Associated Virus Delivery Limitations for Neurological Indications von Benatti, Hector Ribeiro, Gray-Edwards, Heather L

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Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model von Benatti, Hector Ribeiro, Prestigiacomo, Rachel D., Taghian, Toloo, Miller, Rachael, King, Robert, Gounis, Matthew J., Celik, Ugur, Bertrand, Stephanie, Tuominen, Susan, Bierfeldt, Lindsey, Parsley, Elizabeth, Gallagher, Jillian, Hall, Erin F., McElroy, Abigail W., Sena-Esteves, Miguel, Khvorova, Anastasia, Aronin, Neil, Gray-Edwards, Heather L.

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7T MRI Predicts Amelioration of Neurodegeneration in the Brain after AAV Gene Therapy von Gray-Edwards, Heather L., Maguire, Anne S., Salibi, Nouha, Ellis, Lauren E., Voss, Taylor L., Diffie, Elise B., Koehler, Jey, Randle, Ashley N., Taylor, Amanda R., Brunson, Brandon L., Denney, Thomas S., Beyers, Ronald J., Gentry, Atoska S., Gross, Amanda L., Batista, Ana R., Sena-Esteves, Miguel, Martin, Douglas R.

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Real-time MR tracking of AAV gene therapy with βgal-responsive MR probe in a murine model of GM1-gangliosidosis von Taghian, Toloo, Batista, Ana Rita, Kamper, Sarah, Caldwell, Michael, Lilley, Laura, Li, Hao, Rodriguez, Paola, Mesa, Katerina, Zheng, Shaokuan, King, Robert M., Gounis, Matthew J., Todeasa, Sophia, Maguire, Anne, Martin, Douglas R., Sena-Esteves, Miguel, Meade, Thomas J., Gray-Edwards, Heather L.

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Canine Models of Inherited Musculoskeletal and Neurodegenerative Diseases von Story, Brett D, Miller, Matthew E, Bradbury, Allison M, Million, Emily D, Duan, Dongsheng, Taghian, Toloo, Faissler, Dominik, Fernau, Deborah, Beecy, Sidney J, Gray-Edwards, Heather L

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AAV-Mediated Gene Delivery in a Feline Model of Sandhoff Disease Corrects Lysosomal Storage in the Central Nervous System von Rockwell, Hannah E., McCurdy, Victoria J., Eaton, Samuel C., Wilson, Diane U., Johnson, Aime K., Randle, Ashley N., Bradbury, Allison M., Gray-Edwards, Heather L., Baker, Henry J., Hudson, Judith A., Cox, Nancy R., Sena-Esteves, Miguel, Seyfried, Thomas N., Martin, Douglas R.

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AAV gene therapy for Tay-Sachs disease von 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., Martin, Douglas R., Gray-Edwards, Heather L., Sena-Esteves, Miguel

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A comprehensive study of a 29-capsid AAV library in a non-human primate central nervous system von Kondratov, Oleksandr, Kondratova, Liudmyla, Mandel, Ronald J., Coleman, Kirsten, Savage, Michael A., Gray-Edwards, Heather L., Ness, Timothy J., Rodriguez-Lebron, Edgar, Bell, Robert D., Rabinowitz, Joseph, Gamlin, Paul D., Zolotukhin, Sergei

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A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system von 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

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A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna von 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.

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Lipidomic Evaluation of Feline Neurologic Disease after AAV Gene Therapy von Gray-Edwards, Heather L, Jiang, Xuntian, Randle, Ashley N, Taylor, Amanda R, Voss, Taylor L, Johnson, Aime K, McCurdy, Victoria J, Sena-Esteves, Miguel, Ory, Daniel S, Martin, Douglas R

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A minimally invasive endovascular approach to the cerebellopontine angle cistern enables broad CNS biodistribution of scAAV9-CB-GFP von Benatti, Hector Ribeiro, Anagnostakou, Vania, Taghian, Toloo, Hall, Erin F., Nath, Sarah, Heilman, Carl B., Beneduce, Brandon M., Leporati, Anita, Raskett, Christopher, Epshtein, Mark, King, Robert, Gounis, Matthew J., Malek, Adel M., Gray-Edwards, Heather L.

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Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector von Choudhury, Sourav R, Harris, Anne F, Cabral, Damien J, Keeler, Allison M, Sapp, Ellen, Ferreira, Jennifer S, Gray-Edwards, Heather L, Johnson, Jacob A, Johnson, Aime K, Su, Qin, Stoica, Lorelei, DiFiglia, Marian, Aronin, Neil, Martin, Douglas R, Gao, Guangping, Sena-Esteves, Miguel

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