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Complex dietary polysaccharide modulates gut immune function and microbiota, and promotes protection from autoimmune diabetes

Immunology, 2019-05, Vol.157 (1), p.70-85

2019

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Activation of T cell checkpoint pathways during β‐cell antigen presentation by engineered dendritic cells promotes protection from type 1 diabetes

Immunology, 2022-07, Vol.166 (3), p.341-356

2022

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Intrathecal administration of AAV/GALC vectors in 10-11-day-old twitcher mice improves survival and is enhanced by bone marrow transplant

Journal of neuroscience research, 2016-11, Vol.94 (11), p.1138-1151

2016

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pH of Drinking Water Influences the Composition of Gut Microbiome and Type 1 Diabetes Incidence

Diabetes (New York, N.Y.), 2014-02, Vol.63 (2), p.632-644

2014

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Immunological considerations for treating globoid cell leukodystrophy

Journal of neuroscience research, 2016-11, Vol.94 (11), p.1349-1358

2016

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Induction of Innate Immune Response through TLR2 and Dectin 1 Prevents Type 1 Diabetes

The Journal of immunology (1950), 2008-12, Vol.181 (12), p.8323-8334

2008

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Sexually dimorphic patterns in electroencephalography power spectrum and autism-related behaviors in a rat model of fragile X syndrome

Neurobiology of disease, 2020-12, Vol.146, p.105118-105118, Article 105118

2020

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Sex Difference Leads to Differential Gene Expression Patterns and Therapeutic Efficacy in Mucopolysaccharidosis IVA Murine Model Receiving AAV8 Gene Therapy

International journal of molecular sciences, 2022-10, Vol.23 (20), p.12693

2022

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Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease

Human gene therapy, 2016-07, Vol.27 (7), p.509

2016

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Liver-Targeted AAV8 Gene Therapy Ameliorates Skeletal and Cardiovascular Pathology in a Mucopolysaccharidosis IVA Murine Model

Molecular therapy. Methods & clinical development, 2020-09, Vol.18, p.50-61

2020

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Gene therapy using an ortholog of human fragile X mental retardation protein partially rescues behavioral abnormalities and EEG activity

Molecular therapy. Methods & clinical development, 2021-09, Vol.22, p.196-209

2021

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Construction of a hybrid β-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in vivo

Molecular therapy. Methods & clinical development, 2016-01, Vol.3 (C), p.15057-15057, Article 15057

2016

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Bone Marrow Is a Preferential Homing Site for Autoreactive T-Cells in Type 1 Diabetes

Diabetes (New York, N.Y.), 2007-09, Vol.56 (9), p.2251-2259

2007

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Fungal β-glucan, a Dectin-1 ligand, promotes protection from type 1 diabetes by inducing regulatory innate immune response

The Journal of immunology (1950), 2014-10, Vol.193 (7), p.3308-3321

2014

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The presence of CpGs in AAV gene therapy vectors induces a plasmacytoid dendritic cell-like population very early after administration

Cellular immunology, 2024-05, Vol.399-400, p.104823-104823, Article 104823

2024

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Engineered Dendritic Cell-Directed Concurrent Activation of Multiple T cell Inhibitory Pathways Induces Robust Immune Tolerance

Scientific reports, 2019-08, Vol.9 (1), p.12065-16, Article 12065

2019

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Intracisternal administration of AAV9 gene therapies to target the central nervous system

Molecular genetics and metabolism, 2021-02, Vol.132 (2), p.S89-S89

2021

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Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe’s Disease after Global Gene Therapy

Molecular therapy, 2018-03, Vol.26 (3), p.874-889

2018

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TLR2- and Dectin 1-associated innate immune response modulates T-cell response to pancreatic β-cell antigen and prevents type 1 diabetes

Diabetes (New York, N.Y.), 2015-04, Vol.64 (4), p.1341-1357

2015

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Characterization and biodistribution of under-employed gene therapy vector AAV7

Journal of virology, 2023-11, Vol.97 (11), p.e0116323-e0116323

2023

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Treatment of GM2 Gangliosidosis in Adult Sandhoff Mice Using an Intravenous Self-Complementary Hexosaminidase Vector

Current gene therapy, 2022-06, Vol.22 (3), p.262

2022

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551. Enhancement of Gene Therapy Treatment for Sandhoff Disease Through Complimentary Drug Therapy

Molecular therapy, 2016-05, Vol.24, p.S221-S221

2016

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725. Assessment of CSF Route for Gene Delivery in Sandhoff Mice Using AAV9 Expressing an Hexosaminidase Isoenzyme

Molecular therapy, 2016-05, Vol.24, p.S286-S286

2016

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57. Intrathecal Administration of AAV/GALC Vectors in Juvenile Twitcher Mice Improves Survival and Is Enhanced by Bone Marrow Transplant

Molecular therapy, 2016-05, Vol.24, p.S25-S25

2016

Volltextzugriff (PDF)

363. Improvement of Sandhoff Phenotype Following Intravenous Injection of Adeno-Associated Viral Vector Expressing a Hexosaminidase Isoenzyme in Adult Sandhoff Mice: Preclinical Safety and Efficacy Study

Molecular therapy, 2016-05, Vol.24, p.S145-S146

2016

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Complex dietary polysaccharide modulates gut immune function and microbiota, and promotes protection from autoimmune diabetes

Activation of T cell checkpoint pathways during β‐cell antigen presentation by engineered dendritic cells promotes protection from type 1 diabetes

Intrathecal administration of AAV/GALC vectors in 10-11-day-old twitcher mice improves survival and is enhanced by bone marrow transplant

pH of Drinking Water Influences the Composition of Gut Microbiome and Type 1 Diabetes Incidence

Immunological considerations for treating globoid cell leukodystrophy

Induction of Innate Immune Response through TLR2 and Dectin 1 Prevents Type 1 Diabetes

Sexually dimorphic patterns in electroencephalography power spectrum and autism-related behaviors in a rat model of fragile X syndrome

Sex Difference Leads to Differential Gene Expression Patterns and Therapeutic Efficacy in Mucopolysaccharidosis IVA Murine Model Receiving AAV8 Gene Therapy

Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease

Liver-Targeted AAV8 Gene Therapy Ameliorates Skeletal and Cardiovascular Pathology in a Mucopolysaccharidosis IVA Murine Model

Gene therapy using an ortholog of human fragile X mental retardation protein partially rescues behavioral abnormalities and EEG activity

Construction of a hybrid β-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in vivo

Bone Marrow Is a Preferential Homing Site for Autoreactive T-Cells in Type 1 Diabetes

Fungal β-glucan, a Dectin-1 ligand, promotes protection from type 1 diabetes by inducing regulatory innate immune response

The presence of CpGs in AAV gene therapy vectors induces a plasmacytoid dendritic cell-like population very early after administration

Engineered Dendritic Cell-Directed Concurrent Activation of Multiple T cell Inhibitory Pathways Induces Robust Immune Tolerance

Intracisternal administration of AAV9 gene therapies to target the central nervous system

Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe’s Disease after Global Gene Therapy

TLR2- and Dectin 1-associated innate immune response modulates T-cell response to pancreatic β-cell antigen and prevents type 1 diabetes

Characterization and biodistribution of under-employed gene therapy vector AAV7

Treatment of GM2 Gangliosidosis in Adult Sandhoff Mice Using an Intravenous Self-Complementary Hexosaminidase Vector

551. Enhancement of Gene Therapy Treatment for Sandhoff Disease Through Complimentary Drug Therapy

725. Assessment of CSF Route for Gene Delivery in Sandhoff Mice Using AAV9 Expressing an Hexosaminidase Isoenzyme

57. Intrathecal Administration of AAV/GALC Vectors in Juvenile Twitcher Mice Improves Survival and Is Enhanced by Bone Marrow Transplant

363. Improvement of Sandhoff Phenotype Following Intravenous Injection of Adeno-Associated Viral Vector Expressing a Hexosaminidase Isoenzyme in Adult Sandhoff Mice: Preclinical Safety and Efficacy Study

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