Precision medicine using whole genome sequencing in a cat identifies a novel COL5A1 variant for classical Ehlers‐Danlos syndrome

por McElroy, Abigail, Gray‐Edwards, Heather, Coghill, Lyndon M., Lyons, Leslie A.
Publicado 2023

BSLD-01 DYNAMIC CONTROL OF CSF FOR PERSONALIZED CNS DRUG DELIVERY FOR LEPTOMINGEAL DISEASE

por Kumthekar, Priya, Benatti, Hector Ribeiro, Taghian, Toloo, Nagpal, Seema, Gormley, William, Baker, William, Patel, Rajan, Brown, Emma, Glicksman, Marcie, Gray-Edwards, Heather
Publicado 2023

Large Animal Models of Huntington’s Disease: What We Have Learned and Where We Need to Go Next

por Howland, David, Ellederova, Zdenka, Aronin, Neil, Fernau, Deborah, Gallagher, Jill, Taylor, Amanda, Hennebold, Jon, Weiss, Alison R., Gray-Edwards, Heather, McBride, Jodi
Publicado 2020

Natural history study of glycan accumulation in large animal models of GM2 gangliosidoses

por Cavender, Catlyn, Mangini, Linley, Van Vleet, Jeremy L., Corado, Carley, McCullagh, Emma, Gray-Edwards, Heather L., Martin, Douglas R., Crawford, Brett E., Lawrence, Roger
Publicado 2020

Canine Models of Inherited Musculoskeletal and Neurodegenerative Diseases

por 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.
Publicado 2020

Lipidomic Evaluation of Feline Neurologic Disease after AAV Gene Therapy

por 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.
Publicado 2017

AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates

por 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
Publicado 2022

Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease

por McCurdy, Victoria J., Johnson, Aime K., Gray-Edwards, Heather L., Randle, Ashley N., Bradbury, Allison M., Morrison, Nancy E., Hwang, Misako, Baker, Henry J., Cox, Nancy R., Sena-Esteves, Miguel, Martin, Douglas R.
Publicado 2020

PEA15 loss of function and defective cerebral development in the domestic cat

por 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.
Publicado 2020

Brain Alterations in Aged OVT73 Sheep Model of Huntington’s Disease: An MRI Based Approach

por Taghian, Toloo, Gallagher, Jillian, Batcho, Erin, Pullan, Caitlin, Kuchel, Tim, Denney, Thomas, Perumal, Raj, Moore, Shamika, Muirhead, Robb, Herde, Paul, Johns, Daniel, Christou, Chris, Taylor, Amanda, Passler, Thomas, Pulaparthi, Sanjana, Hall, Erin, Chandra, Sundeep, O’Neill, Charles A., Gray-Edwards, Heather
Publicado 2022

Real-time MR tracking of AAV gene therapy with βgal-responsive MR probe in a murine model of GM1-gangliosidosis

por 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.
Publicado 2021

A pentasaccharide for monitoring pharmacodynamic response to gene therapy in GM1 gangliosidosis

por Kell, Pamela, Sidhu, Rohini, Qian, Mingxing, Mishra, Sonali, Nicoli, Elena-Raluca, D'Souza, Precilla, Tifft, Cynthia J., Gross, Amanda L., Gray-Edwards, Heather L., Martin, Douglas R., Sena- Esteves, Miguel, Dietzen, Dennis J., Singh, Manmilan, Luo, Jingqin, Schaffer, Jean E., Ory, Daniel S., Jiang, Xuntian
Publicado 2023

Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation

por 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
Publicado 2023

Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model

por 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.
Publicado 2023

AAV-Mediated Gene Delivery in a Feline Model of Sandhoff Disease Corrects Lysosomal Storage in the Central Nervous System

por 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.
Publicado 2015

7T MRI Predicts Amelioration of Neurodegeneration in the Brain after AAV Gene Therapy

por 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.
Publicado 2019

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

por Davidson, Beverly L., Gao, Guangping, Berry-Kravis, Elizabeth, Bradbury, Allison M., Bönnemann, 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
Publicado 2022

Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep

por Ferguson, Chantal M., Godinho, Bruno M.D.C., Alterman, Julia F., Coles, Andrew H., Hassler, Matthew, Echeverria, Dimas, Gilbert, James W., Knox, Emily G., Caiazzi, Jillian, Haraszti, Reka A., King, Robert M., Taghian, Toloo, Puri, Ajit, Moser, Richard P., Gounis, Matthew J., Aronin, Neil, Gray-Edwards, Heather, Khvorova, Anastasia
Publicado 2021

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

por 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
Publicado 2019