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Krabbe disease successfully treated via monotherapy of intrathecal gene therapy

The Journal of clinical investigation, 2020-09, Vol.130 (9), p.4906-4920

2020

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Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals

Human gene therapy, 2018-01, Vol.29 (1), p.15

2018

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Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease

Science translational medicine, 2015-02, Vol.7 (276), p.276ra26

2015

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Forecasting seizures in dogs with naturally occurring epilepsy

PloS one, 2014-01, Vol.9 (1), p.e81920-e81920

2014

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Efficacy and Safety of a Krabbe Disease Gene Therapy

Human gene therapy, 2022-05, Vol.33 (9-10), p.499

2022

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Global CNS correction in a large brain model of human alpha-mannosidosis by intravascular gene therapy

Brain (London, England : 1878), 2020-07, Vol.143 (7), p.2058-2072

2020

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N-acyl-O-phosphocholineserines: structures of a novel class of lipids that are biomarkers for Niemann-Pick C1 disease

Journal of lipid research, 2019-08, Vol.60 (8), p.1410-1424

2019

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Application of N-palmitoyl-O-phosphocholineserine for diagnosis and assessment of response to treatment in Niemann-Pick type C disease

Molecular genetics and metabolism, 2020-04, Vol.129 (4), p.292-302

2020

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Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII

Molecular therapy, 2016-02, Vol.24 (2), p.206-216

2016

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A digital atlas of the dog brain

PloS one, 2012-12, Vol.7 (12), p.e52140-e52140

2012

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A Homozygous MAN2B1 Missense Mutation in a Doberman Pinscher Dog with Neurodegeneration, Cytoplasmic Vacuoles, Autofluorescent Storage Granules, and an α-Mannosidase Deficiency

Genes, 2023-08, Vol.14 (9), p.1746

2023

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Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors

Molecular therapy. Methods & clinical development, 2021-03, Vol.20, p.247-257

2021

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Clinical Improvement of Alpha-mannosidosis Cat Following a Single Cisterna Magna Infusion of AAV1

Molecular therapy, 2016-01, Vol.24 (1), p.26-33

2016

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Pulmonary abnormalities in animal models due to Niemann-Pick type C1 (NPC1) or C2 (NPC2) disease

PloS one, 2013-07, Vol.8 (7), p.e67084-e67084

2013

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Identification of the rostral migratory stream in the canine and feline brain

PloS one, 2012-05, Vol.7 (5), p.e36016-e36016

2012

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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

Human molecular genetics, 2019-10, Vol.28 (R1), p.R119-R131

2019

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Electrodiagnostic Testing and Histopathologic Changes Confirm Peripheral Nervous System Myelin Abnormalities in the Feline Model of Niemann-Pick Disease Type C

Journal of neuropathology and experimental neurology, 2013-03, Vol.72 (3), p.256-262

2013

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Intraventricular Brain Injection of Adeno-Associated Virus Type 1 (AAV1) in Neonatal Mice Results in Complementary Patterns of Neuronal Transduction to AAV2 and Total Long-Term Correction of Storage Lesions in the Brains of β-Glucuronidase-Deficient Mice

Journal of Virology, 2003-06, Vol.77 (12), p.7034-7040

2003

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Aging in the Canine and Feline Brain

The Veterinary clinics of North America. Small animal practice, 2014-11, Vol.44 (6), p.1113-1129

2014

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Tumors affecting the spinal cord of cats: 85 cases (1980-2005)

Journal of the American Veterinary Medical Association, 2008-01, Vol.232 (2), p.237

2008

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Gene therapy for lysosomal storage diseases: the lessons and promise of animal models

The journal of gene medicine, 2004-05, Vol.6 (5), p.481-506

2004

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Biodistribution of Adeno-Associated Virus Gene Therapy Following Cerebrospinal Fluid-Directed Administration

Human gene therapy, 2023-02, Vol.34 (3-4), p.94

2023

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Biodistribution and pharmacodynamics of recombinant human alpha- l-iduronidase (rhIDU) in mucopolysaccharidosis type I-affected cats following multiple intrathecal administrations

Molecular genetics and metabolism, 2011-07, Vol.103 (3), p.268-274

2011

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2-Hydroxypropyl-β-Cyclodextrin Raises Hearing Threshold in Normal Cats and in Cats With Niemann-Pick Type C Disease

Pediatric research, 2010-07, Vol.68 (1), p.52-56

2010

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Krabbe disease successfully treated via monotherapy of intrathecal gene therapy

Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals

Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease

Forecasting seizures in dogs with naturally occurring epilepsy

Efficacy and Safety of a Krabbe Disease Gene Therapy

Global CNS correction in a large brain model of human alpha-mannosidosis by intravascular gene therapy

N-acyl-O-phosphocholineserines: structures of a novel class of lipids that are biomarkers for Niemann-Pick C1 disease

Application of N-palmitoyl-O-phosphocholineserine for diagnosis and assessment of response to treatment in Niemann-Pick type C disease

Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII

A digital atlas of the dog brain

A Homozygous MAN2B1 Missense Mutation in a Doberman Pinscher Dog with Neurodegeneration, Cytoplasmic Vacuoles, Autofluorescent Storage Granules, and an α-Mannosidase Deficiency

Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors

Clinical Improvement of Alpha-mannosidosis Cat Following a Single Cisterna Magna Infusion of AAV1

Pulmonary abnormalities in animal models due to Niemann-Pick type C1 (NPC1) or C2 (NPC2) disease

Identification of the rostral migratory stream in the canine and feline brain

Large animal models contribute to the development of therapies for central and peripheral nervous system dysfunction in patients with lysosomal storage diseases

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial

Electrodiagnostic Testing and Histopathologic Changes Confirm Peripheral Nervous System Myelin Abnormalities in the Feline Model of Niemann-Pick Disease Type C

Intraventricular Brain Injection of Adeno-Associated Virus Type 1 (AAV1) in Neonatal Mice Results in Complementary Patterns of Neuronal Transduction to AAV2 and Total Long-Term Correction of Storage Lesions in the Brains of β-Glucuronidase-Deficient Mice

Aging in the Canine and Feline Brain

Tumors affecting the spinal cord of cats: 85 cases (1980-2005)

Gene therapy for lysosomal storage diseases: the lessons and promise of animal models

Biodistribution of Adeno-Associated Virus Gene Therapy Following Cerebrospinal Fluid-Directed Administration

Biodistribution and pharmacodynamics of recombinant human alpha- l-iduronidase (rhIDU) in mucopolysaccharidosis type I-affected cats following multiple intrathecal administrations

2-Hydroxypropyl-β-Cyclodextrin Raises Hearing Threshold in Normal Cats and in Cats With Niemann-Pick Type C Disease

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