Publications by authors named "Charles Vite"

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Adeno-associated vector corneal gene therapy reverses corneal clouding in a feline model of mucopolysaccharidosis VI.
Brian C Gilger, Liujiang Song, Tomoko Hasegawa, Jacklyn H Salmon, Jacquelyn J Bower, Charles Vite

PLoS One· December 2025

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Effect of disease progression on CSF-directed AAV gene therapy in a large brain animal model of lysosomal storage disease.
Jacqueline E Hunter, Caitlyn M Molony, Wojciech K Panek, Charles H Vite, Sanjeev Chawla

Mol Ther Methods Clin Dev· September 2025

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Intracisternal vs intraventricular injection of AAV1 result in comparable, widespread transduction of the dog brain.
Jacqueline E Hunter, Charles H Vite, Caitlyn M Molony, Patricia A O'Donnell, John H Wolfe

Gene Ther· May 2025

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Accumulation of alkyl-lysophosphatidylcholines in Niemann-Pick disease type C1.
Sonali Mishra, Pamela Kell, David Scherrer, Dennis J Dietzen, Charles H Vite

J Lipid Res· August 2024

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Widespread correction of brain pathology in feline alpha-mannosidosis by dose escalation of intracisternal AAV vector injection.
Jacqueline E Hunter, Caitlyn M Molony, Jessica H Bagel, Patricia O'Donnell, Charles H Vite

Mol Ther Methods Clin Dev· June 2024

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Beta-mannosidosis in a domestic cat associated with a missense variant in MANBA.
Martin L Katz, James Cook, Charles H Vite, Rebecca S Campbell, Lyndon M Coghill

Gene· January 2024

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Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy.
Allison M Bradbury, Jessica Bagel, Gary Swain, Keiko Miyadera, Jill P Pesayco, Charles H Vite

Mol Ther· January 2024

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A Homozygous Missense Mutation in a Doberman Pinscher Dog with Neurodegeneration, Cytoplasmic Vacuoles, Autofluorescent Storage Granules, and an α-Mannosidase Deficiency.
Garrett Bullock, Gary S Johnson, Savannah G Pattridge, Tendai Mhlanga-Mutangadura, Juyuan Guo, Charles H Vite

Genes (Basel)· August 2023

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Biodistribution of Adeno-Associated Virus Gene Therapy Following Cerebrospinal Fluid-Directed Administration.
Xin Chen, Daniel A Lim, Michael W Lawlor, David Dimmock, Charles H Vite

Hum Gene Ther· February 2023

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Efficacy and Safety of a Krabbe Disease Gene Therapy.
Juliette Hordeaux, Brianne A Jeffrey, Jinlong Jian, Gourav R Choudhury, Kristofer Michalson, Charles H Vite

Hum Gene Ther· May 2022

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Meningeal B Cell Clusters Correlate with Submeningeal Pathology in a Natural Model of Multiple Sclerosis.
Molly E Church, Guadalupe Ceja, Megan McGeehan, Miles C Miller, Priscilla Farias, Charles H Vite

J Immunol· July 2021

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Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors.
Rita Ferla, Marialuisa Alliegro, Margherita Dell'Anno, Edoardo Nusco, John M Cullen, Charles H Vite

Mol Ther Methods Clin Dev· March 2021

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Krabbe disease successfully treated via monotherapy of intrathecal gene therapy.
Allison M Bradbury, Jessica H Bagel, Duc Nguyen, Erik A Lykken, Jill Pesayco Salvador, Charles H Vite

J Clin Invest· September 2020

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Global CNS correction in a large brain model of human alpha-mannosidosis by intravascular gene therapy.
Sea Young Yoon, Jacqueline E Hunter, Sanjeev Chawla, Dana L Clarke, Caitlyn Molony, Charles H Vite

Brain· July 2020

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Pharmacokinetics and distribution of 2-hydroxypropyl-β-cyclodextrin following a single intrathecal dose to cats.
Mark L Kao, Susan Stellar, Eric Solon, Alfred Lordi, Nicole Kasica, Charles H Vite

J Inherit Metab Dis· May 2020

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Application of N-palmitoyl-O-phosphocholineserine for diagnosis and assessment of response to treatment in Niemann-Pick type C disease.
Rohini Sidhu, Pamela Kell, Dennis J Dietzen, Nicole Y Farhat, An Ngoc Dang Do, Charles H Vite

Mol Genet Metab· April 2020

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Cerebrospinal fluid and serum glycosphingolipid biomarkers in canine globoid cell leukodystrophy (Krabbe Disease).
Carley R Corado, Jason Pinkstaff, Xuntian Jiang, Evelyn M Galban, Samantha J Fisher, Charles H Vite

Mol Cell Neurosci· January 2020

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Large animal models contribute to the development of therapies for central and peripheral nervous system dysfunction in patients with lysosomal storage diseases.
Brittney L Gurda, Charles H Vite

Hum Mol Genet· October 2019

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-acyl--phosphocholineserines: structures of a novel class of lipids that are biomarkers for Niemann-Pick C1 disease.
Rohini Sidhu, Yawo Mondjinou, Mingxing Qian, Haowei Song, Arun Babu Kumar, Charles H Vite

J Lipid Res· August 2019

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A cross-species approach to disorders affecting brain and behaviour.
Orrin Devinsky, Jordyn M Boesch, Sofia Cerda-Gonzalez, Barbara Coffey, Kathryn Davis, Charles Vite

Nat Rev Neurol· November 2018

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AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease).
Allison M Bradbury, Mohammed A Rafi, Jessica H Bagel, Becky K Brisson, Michael S Marshall, Charles H Vite

Hum Gene Ther· July 2018

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Quantitative DTI metrics in a canine model of Krabbe disease: comparisons versus age-matched controls across multiple ages.
Jonathan Y Li, Dana M Middleton, Steven Chen, Leonard White, Carley R Corado, Charles Vite

Neuroradiol J· April 2018

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LC3 Immunostaining in the Inferior Olivary Nuclei of Cats With Niemann-Pick Disease Type C1 Is Associated With Patterned Purkinje Cell Loss.
Brittney L Gurda, Jessica H Bagel, Samantha J Fisher, Mark L Schultz, Andrew P Lieberman, Charles H Vite

J Neuropathol Exp Neurol· March 2018

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Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals.
Christian Hinderer, Peter Bell, Nathan Katz, Charles H Vite, Jean-Pierre Louboutin

Hum Gene Ther· January 2018

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Canine and Feline Models of Human Genetic Diseases and Their Contributions to Advancing Clinical Therapies
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Brittney L Gurda, Allison M Bradbury, Charles H Vite

Yale J Biol Med· September 2017

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