Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy-Reference-Cited by-同舟云学术

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Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy

Published:2023-12 Issue: Volume:31 Page:101127
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Ellison Stuart,Liao Aiyin,Gleitz Hélène F.E.,Parker Helen,Booth Laura,Robinson John,Wood Shaun,Taylor Jessica,Holley Rebecca,Bigger Brian W.

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference46 articles.

1. Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome);Martin;Pediatrics,2008

2. Mucopolysaccharidosis Type II;Scarpa,1993

3. Advances in therapies for neurological Lysosomal Storage Disorders;Ellison;J. Inherit. Metab. Dis.,2023

4. Epidemiology of mucopolysaccharidoses;Khan;Mol. Genet. Metabol.,2017

5. Intravenous Enzyme Replacement Therapy in Mucopolysaccharidoses: Clinical Effectiveness and Limitations;Parini;Int. J. Mol. Sci.,2020

Cited by 1 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. Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy;Molecular Therapy - Methods & Clinical Development;2024-06

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