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Regulation of BMP4/Dpp retrotranslocation and signaling by deglycosylation

eLife, 2020-07, Vol.9

2020

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Efficacy of syngeneic bone marrow transplant for the treatment of multiple sulfatase deficiency

Molecular genetics and metabolism, 2023-02, Vol.138 (2), p.107278, Article 107278

2023

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Changes in food consumption from an agricultural-based economy to industrialisation: Uruguay (1900–70)

Rural history, 2023-10, Vol.34 (2), p.220-242

2023

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AAV9-mediated FIG4 delivery prolongs life span in Charcot-Marie-Tooth disease type 4J mouse model

The Journal of clinical investigation, 2021-06, Vol.131 (11), p.1-11

2021

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Nfkbid overexpression in NOD mice elicits complete type 1 diabetes resistance in part associated with enhanced thymic deletion of pathogenic CD8 T-cells and increased numbers and activity of regulatory T-cells.1,2,3

The Journal of immunology (1950), 2022-06, Vol.209 (2), p.227-237

2022

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Nfkbid Overexpression in Nonobese Diabetic Mice Elicits Complete Type 1 Diabetes Resistance in Part Associated with Enhanced Thymic Deletion of Pathogenic CD8 T Cells and Increased Numbers and Activity of Regulatory T Cells

The Journal of immunology (1950), 2022-07, Vol.209 (2), p.227-237

2022

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Efficacy of a scAAV9/ viral vector for the treatment of multiple sulfatase deficiency

Molecular genetics and metabolism, 2022-02, Vol.135 (2), p.S101

2022

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Efficacy of a scAAV9/SUMF1 viral vector for the treatment of multiple sulfatase deficiency

Molecular genetics and metabolism, 2022-02, Vol.135 (2), p.S101-S101

2022

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Nfkbid overexpression in NOD mice elicits complete type 1 diabetes resistance

The Journal of immunology (1950), 2019-05, Vol.202 (1_Supplement), p.115-115.8

2019

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Natural history of GM1 gangliosidosis mouse models generated by The Jackson Laboratory Rare and Orphan Disease Center

Molecular genetics and metabolism, 2020-02, Vol.129 (2), p.S46-S46

2020

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Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency

Molecular therapy, 2024-08

2024

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Preclinical studies to support the intrathecal delivery of scAAV9/SUMF1 as a gene replacement therapy for multiple sulfatase deficiency

Molecular genetics and metabolism, 2021-02, Vol.132 (2), p.S17-S18

2021

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New mouse models with hypomorphic SUMF1 variants mimic attenuated forms of multiple sulfatase deficiency

Journal of inherited metabolic disease, 2023-03, Vol.46 (2), p.335-347

2023

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Treatment of T1D via optimized expansion of antigen-specific Tregs induced by IL-2/anti-IL-2 monoclonal antibody complexes and peptide/MHC tetramers

Scientific reports, 2018-05, Vol.8 (1), p.8106-14, Article 8106

2018

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A Hypermorphic Nfkbid Allele Contributes to Impaired Thymic Deletion of Autoreactive Diabetogenic CD8 + T Cells in NOD Mice

The Journal of immunology (1950), 2018-10, Vol.201 (7), p.1907-1917

2018

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A hypermorphic Nfkbid allele contributes to impaired thymic deletion of autoreactive diabetogenic CD8+ T-cells in NOD mice1,2

The Journal of immunology (1950), 2018-08, Vol.201 (7), p.1907-1917

2018

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In vivo detection of peripherin-specific autoreactive B cells during type 1 diabetes pathogenesis

The Journal of immunology (1950), 2014-04, Vol.192 (7), p.3080-3090

2014

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The presence and preferential activation of Tregs diminishes adoptive transfer of autoimmune diabetes by polyclonal NOD T cell effectors into NSG versus NOD-scid mice1

The Journal of immunology (1950), 2015-08, Vol.195 (7), p.3011-3019

2015

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The Presence and Preferential Activation of Regulatory T Cells Diminish Adoptive Transfer of Autoimmune Diabetes by Polyclonal Nonobese Diabetic (NOD) T Cell Effectors into NSG versus NOD-scid Mice

The Journal of immunology (1950), 2015-10, Vol.195 (7), p.3011-3019

2015

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Cholera toxin subunit B peptide fusion proteins reveal impaired oral tolerance induction in diabetes‐prone but not in diabetes‐resistant mice

European journal of immunology, 2013-11, Vol.43 (11), p.2969-2979

2013

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A locus on mouse chromosome 13 inversely regulates CD1d expression and the development of invariant natural killer T-cells

Genes and immunity, 2015-04, Vol.16 (3), p.221-230

2015

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Impaired Oral Tolerance Induction in Diabetes Prone but not in Diabetes Resistant Mice Revealed by Cholera Toxin Subunit B-Peptide Fusion Proteins

European journal of immunology, 2013-08, Vol.43 (11)

2013

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In vivo detection of peripherin-specific autoreactive B cells during type 1 diabetes pathogenesis1

The Journal of immunology (1950), 2014-03, Vol.192 (7), p.3080-3090

2014

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Targeting of a T cell agonist peptide to lysosomes by DNA vaccination induces tolerance in the nonobese diabetic mouse

The Journal of immunology (1950), 2011-04, Vol.186 (7), p.4078-4087

2011

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Regulation of BMP4/Dpp retrotranslocation and signaling by deglycosylation

Efficacy of syngeneic bone marrow transplant for the treatment of multiple sulfatase deficiency

Changes in food consumption from an agricultural-based economy to industrialisation: Uruguay (1900–70)

AAV9-mediated FIG4 delivery prolongs life span in Charcot-Marie-Tooth disease type 4J mouse model

Nfkbid overexpression in NOD mice elicits complete type 1 diabetes resistance in part associated with enhanced thymic deletion of pathogenic CD8 T-cells and increased numbers and activity of regulatory T-cells.1,2,3

Nfkbid Overexpression in Nonobese Diabetic Mice Elicits Complete Type 1 Diabetes Resistance in Part Associated with Enhanced Thymic Deletion of Pathogenic CD8 T Cells and Increased Numbers and Activity of Regulatory T Cells

Efficacy of a scAAV9/ viral vector for the treatment of multiple sulfatase deficiency

Efficacy of a scAAV9/SUMF1 viral vector for the treatment of multiple sulfatase deficiency

Nfkbid overexpression in NOD mice elicits complete type 1 diabetes resistance

Natural history of GM1 gangliosidosis mouse models generated by The Jackson Laboratory Rare and Orphan Disease Center

Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency

Preclinical studies to support the intrathecal delivery of scAAV9/SUMF1 as a gene replacement therapy for multiple sulfatase deficiency

New mouse models with hypomorphic SUMF1 variants mimic attenuated forms of multiple sulfatase deficiency

Stathmin-2 loss leads to neurofilament-dependent axonal collapse driving motor and sensory denervation

Treatment of T1D via optimized expansion of antigen-specific Tregs induced by IL-2/anti-IL-2 monoclonal antibody complexes and peptide/MHC tetramers

A Hypermorphic Nfkbid Allele Contributes to Impaired Thymic Deletion of Autoreactive Diabetogenic CD8 + T Cells in NOD Mice

A hypermorphic Nfkbid allele contributes to impaired thymic deletion of autoreactive diabetogenic CD8+ T-cells in NOD mice1,2

In vivo detection of peripherin-specific autoreactive B cells during type 1 diabetes pathogenesis

The presence and preferential activation of Tregs diminishes adoptive transfer of autoimmune diabetes by polyclonal NOD T cell effectors into NSG versus NOD-scid mice1

The Presence and Preferential Activation of Regulatory T Cells Diminish Adoptive Transfer of Autoimmune Diabetes by Polyclonal Nonobese Diabetic (NOD) T Cell Effectors into NSG versus NOD-scid Mice

Cholera toxin subunit B peptide fusion proteins reveal impaired oral tolerance induction in diabetes‐prone but not in diabetes‐resistant mice

A locus on mouse chromosome 13 inversely regulates CD1d expression and the development of invariant natural killer T-cells

Impaired Oral Tolerance Induction in Diabetes Prone but not in Diabetes Resistant Mice Revealed by Cholera Toxin Subunit B-Peptide Fusion Proteins

In vivo detection of peripherin-specific autoreactive B cells during type 1 diabetes pathogenesis1

Targeting of a T cell agonist peptide to lysosomes by DNA vaccination induces tolerance in the nonobese diabetic mouse

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