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Mucopolysaccharidoses type I gene therapy

Published:2021-07-09 Issue:5 Volume:44 Page:1088-1098
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:Jrnl of Inher Metab Disea

Author:

Hurt Sarah C.¹²,Dickson Patricia I.²³,Curiel David T.¹^ORCID

Affiliation:

1. Cancer Biology Division, Department of Radiation Oncology Washington University School of Medicine St. Louis Missouri USA

2. Department of Genetics Washington University School of Medicine St. Louis Missouri USA

3. Department of Pediatrics Washington University School of Medicine St. Louis Missouri USA

Funder

National Institutes of Health

National Science Foundation

Publisher

Wiley

Subject

Genetics(clinical),Genetics

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/jimd.12414

Reference77 articles.

1. Current Progress in Therapeutic Gene Editing for Monogenic Diseases

2. The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK

3. Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry

4. Diversity of mutations and distribution of single nucleotide polymorphic alleles in the human α-l-iduronidase (IDUA) gene

5. Enzyme-Replacement Therapy in Mucopolysaccharidosis I

Cited by 6 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Combining angiotensin receptor blockade and enzyme replacement therapy for vascular disease in mucopolysaccharidosis type I;Molecular Genetics and Metabolism Reports;2024-03

2. Growth and Related Treatment Factors in Mucopolysaccharidoses Type I and II: A Systematic Review;Journal of Pediatrics Review;2023-10-01

3. In vivo editing of the pan-endothelium by immunity evading simian adenoviral vector;Biomedicine & Pharmacotherapy;2023-02

4. Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction;Human Gene Therapy;2023-01-01

5. Gene editing strategies to treat lysosomal disorders: The example of mucopolysaccharidoses;Advanced Drug Delivery Reviews;2022-12

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