Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches-Reference-Cited by-同舟云学术

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Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches

Published:2018-11 Issue:S2 Volume:44 Page:
ISSN:1824-7288
Container-title:Italian Journal of Pediatrics
language:en
Short-container-title:Ital J Pediatr

Author:

Fraldi Alessandro,Serafini Marta,Sorrentino Nicolina Cristina,Gentner Bernhard,Aiuti Alessandro,Bernardo Maria Ester

Publisher

Springer Science and Business Media LLC

Subject

General Medicine

Link

http://link.springer.com/content/pdf/10.1186/s13052-018-0565-y.pdf

Reference68 articles.

1. Neufeld EF, Muenzer J. The metabolic and molecular bases of inherited disease. New York: McGraw-Hill; 2001.

2. Clarke LA. The mucopolysaccharidoses: a success of molecular medicine. Expert Rev Mol Med. 2008;10:e1.

3. Neufeld EF, Fratantoni JC. Inborn errors of mucopolysaccharide metabolism. Science. 1970;169:141–6.

4. Valayannopoulos V, Wijburg FA. Therapy for the mucopolysaccharidoses. Rheumatology (Oxford). 2011;50:v49–59.

5. Noh H, Lee JI. Current and potential therapeutic strategies for mucopolysaccharidoses. J Clin Pharm Ther. 2014;39:215–24.

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3. An empowered, clinically viable hematopoietic stem cell gene therapy for the treatment of multisystemic mucopolysaccharidosis type II;Molecular Therapy;2024-03

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

5. Mucopolysaccharidosis type I Hurler-Scheie syndrome: a case report;Annals of Medicine & Surgery;2023-11-27

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