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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.15-24

2018

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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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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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Efficacy and Safety of a Krabbe Disease Gene Therapy

Human gene therapy, 2022-05, Vol.33 (9-10), p.499-517

2022

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Accumulation of alkyl-lysophosphatidylcholines in Niemann-Pick disease type C1

Journal of lipid research, 2024-08, Vol.65 (8), p.100600, Article 100600

2024

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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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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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AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease)

Human gene therapy, 2018-07, Vol.29 (7), p.785-801

2018

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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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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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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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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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Widespread correction of brain pathology in feline alpha-mannosidosis by dose escalation of intracisternal AAV vector injection

Molecular therapy. Methods & clinical development, 2024-06, Vol.32 (2), p.101272, Article 101272

2024

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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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Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna

Molecular therapy. Methods & clinical development, 2014-01, Vol.1 (C), p.14051-14051, Article 14051

2014

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

The Lancet (British edition), 2017-10, Vol.390 (10104), p.1758-1768

2017

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

Human gene therapy, 2023-02, Vol.34 (3-4), p.94-111

2023

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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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Krabbe disease successfully treated via monotherapy of intrathecal gene therapy

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

Forecasting seizures in dogs with naturally occurring epilepsy

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

Efficacy and Safety of a Krabbe Disease Gene Therapy

Accumulation of alkyl-lysophosphatidylcholines in Niemann-Pick disease type C1

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

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

AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe 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

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

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

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

Widespread correction of brain pathology in feline alpha-mannosidosis by dose escalation of intracisternal AAV vector injection

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

Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna

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

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

Aging in the Canine and Feline Brain

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