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Gene therapy for Mucopolysaccharidoses

Published:2018-02 Issue:2 Volume:123 Page:59-68
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Sawamoto Kazuki,Chen Hui-Hsuan,Alméciga-Díaz Carlos J.,Mason Robert W.,Tomatsu Shunji

Funder

National MPS Society

Austrian MPS Society

International Morquio Organization

Carol Ann Foundation

Japanese MPS Family Society

National Institutes of Health

NIH

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference102 articles.

1. The mucopolysaccharidoses;Neufeld;The Metabolic and Molecular Bases of Inherited Disease,2001

2. Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VI;Turbeville;Mol. Genet. Metab.,2011

3. Hematopoietic cell transplantation for mucopolysaccharidosis patients is safe and effective: results after implementation of international guidelines;Aldenhoven;Biol. Blood Marrow Transplant,2015

4. Allogeneic hematopoietic stem cell transplantation in thirty-four pediatric cases of mucopolysaccharidosis-a ten-year report from the China children transplant group;Wang;Biol. Blood Marrow Transplant,2016

5. Hematopoietic stem cell transplantation for Morquio A syndrome;Yabe;Mol. Genet. Metab.,2016

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1. Foramen magnum stenosis in patients with mucopolysaccharidoses: diagnosis and surgical treatment. Review of literature;Russian Journal for Personalized Medicine;2024-06-11

2. Adeno-associated virus-based gene therapy delivering combinations of two growth-associated genes to MPS IVA mice;Molecular Therapy - Nucleic Acids;2024-06

3. Molecular Mechanisms in Pathophysiology of Mucopolysaccharidosis and Prospects for Innovative Therapy;International Journal of Molecular Sciences;2024-01-17

4. Principles of novel cellular and molecular cardiovascular therapeutics;Genomic and Molecular Cardiovascular Medicine;2024

5. Iron oxide-coupled CRISPR-nCas9-based genome editing assessment in mucopolysaccharidosis IVA mice;Molecular Therapy - Methods & Clinical Development;2023-12

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