Targeting the Root Cause of Mucopolysaccharidosis IIIA with a New scAAV9 Gene Replacement Vector-Reference-Cited by-同舟云学术

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Targeting the Root Cause of Mucopolysaccharidosis IIIA with a New scAAV9 Gene Replacement Vector

Published:2020-12 Issue: Volume:19 Page:474-485
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Bobo Tierra A.,Samowitz Preston N.,Robinson Michael I.,Fu Haiyan

Funder

Aislinne’s Wish Foundation

Abby Grace Foundation

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference54 articles.

1. The mucopolysaccharidoses;Neufeld,2001

2. Human liver sulphamate sulphohydrolase. Determinations of native protein and subunit Mr values and influence of substrate agylcone structure on catalytic properties;Freeman;Biochem. J.,1986

3. Molecular genetics of mucopolysaccharidosis type IIIA and IIIB: Diagnostic, clinical, and biological implications;Yogalingam;Hum. Mutat.,2001

4. Sanfilippo syndrome: a mini-review;Valstar;J. Inherit. Metab. Dis.,2008

5. Gene therapy using adeno-associated virus vectors;Daya;Clin. Microbiol. Rev.,2008

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