Shunji Tomatsu

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Professor

Nemours Children's Health System

United States

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About

Shunji Tomatsu is a Professor at Nemours Children’s Health in the United States. His research focuses on gene therapy and cell-free therapies for lysosomal storage disorders, particularly mucopolysaccharidosis IVA. Recent publications include studies on lysosomal-mitochondrial interactions, lentiviral vectors, and the use of CRISPR/Cas9 for gene editing applications.

Recent Grants

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Newborn Screening and Biomarkers for Mucopolysaccharidoses

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Oral tolerance in enzyme replacement therapy of Morquio A disease

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Non-invasive functional assessment and pathogenesis of Morquio A

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AAV Gene Therapy for Mucopolysaccharidosis IVA

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2022

Collaborators (4)

Yojiro Yamanaka

Associate Professor

McGill University

CANADA

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Zhengyu Ma

Research Scientist/Head of T Cell Immunology Lab

Novartis Institutes for BioMedical Research Inc

UNITED STATES

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Antoine Caillon

Assistant Professor

University of Montreal

CANADA

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María L. Couce

SPAIN

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Shunji Tomatsu

Research Interests

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About

Recent Grants

Articles (26)

Three-dimensional human mucopolysaccharidosis IVA chondrocyte culture reveals significant impairments in the lysosomal-mitochondrial crosstalk

Assessment of different promoters in lentiviral vectors for expression of the N-acetyl-galactosamine-6-sulfate sulfatase gene

Integrase-Deficient Lentiviral Vector as a Platform for Efficient CRISPR/Cas9-Mediated Gene Editing for Mucopolysaccharidosis IVA

Mesenchymal Stem Cell-Derived Extracellular Vesicles: Seeking into Cell-Free Therapies for Bone-Affected Lysosomal Storage Disorders

Identification of Surrogate Biomarkers for Mucopolysaccharidosis Type IVA

CRISPR/nCas9-Edited CD34+ Cells Rescue Mucopolysaccharidosis IVA Fibroblasts Phenotype

AAV gene therapy for mucopolysaccharidoses

Potential Targeting Mechanisms for Bone-Directed Therapies

Adeno-associated virus-based gene therapy delivering combinations of two growth-associated genes to MPS IVA mice

Heterologous HSPC Transplantation Rescues Neuroinflammation and Ameliorates Peripheral Manifestations in the Mouse Model of Lysosomal Transmembrane Enzyme Deficiency, MPS IIIC

Morquio A Syndrome: Identification of Differential Patterns of Molecular Pathway Interactions in Bone Lesions

Current Strategies for Increasing Knock-In Efficiency in CRISPR/Cas9-Based Approaches

Molecular Mechanisms in Pathophysiology of Mucopolysaccharidosis and Prospects for Innovative Therapy

Adeno-Associated Virus Gene Transfer Ameliorates Progression of Skeletal Lesions in Mucopolysaccharidosis IVA Mice

Lentiviral Vector-Mediated Ex Vivo Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis IVA Murine Model

Bone Growth Induction in Mucopolysaccharidosis IVA Mouse

Detection of Glycosaminoglycans in Biological Specimens

The landscape of CRISPR/Cas9 for inborn errors of metabolism

Mucopolysaccharidosis IVA: Current Disease Models and Drawbacks

Mucopolysaccharidoses: Cellular Consequences of Glycosaminoglycans Accumulation and Potential Targets

A Roadmap for Potential Improvement of Newborn Screening for Inherited Metabolic Diseases Following Recent Developments and Successful Applications of Bivariate Normal Limits for Pre-Symptomatic Detection of MPS I, Pompe Disease, and Krabbe Disease

Regulation of Molecular Targets in Osteosarcoma Treatment

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

Molecular Mechanism of Induction of Bone Growth by the C-Type Natriuretic Peptide

Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases

Glucosamine amends CNS pathology in mucopolysaccharidosis IIIC mouse expressing misfolded HGSNAT

Collaborators (4)

Yojiro Yamanaka

Zhengyu Ma

Antoine Caillon

María L. Couce

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