Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study-Reference-Cited by-同舟云学术

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Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study

Published:2024-03 Issue:1 Volume:32 Page:101168
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Ryckman Alex E.^ORCID,Deschenes Natalie M.,Quinville Brianna M.,Osmon Karlaina J.L.,Mitchell Melissa,Chen Zhilin,Gray Steven J.,Walia Jagdeep S.

Funder

GlycoNet

Cyrus Tang Foundation

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference61 articles.

1. Partial enzyme deficiencies: residual activities and the development of neurological disorders;Conzelmann;Dev. Neurosci.,1983

2. Improved outcome of N-butyldeoxygalactonojirimycin-mediated substrate reduction therapy in a mouse model of Sandhoff disease;Andersson;Neurobiol. Dis.,2004

3. Delayed symptom onset and increased life expectancy in Sandhoff disease mice treated with N-butyldeoxynojirimycin;Jeyakumar;Proc. Natl. Acad. Sci. USA,1999

4. Chaperone therapy for GM2 gangliosidosis: effects of pyrimethamine on β-hexosaminidase activity in Sandhoff fibroblasts;Chiricozzi;Mol. Neurobiol.,2014

5. pyrimethamine as a potential pharmacological chaperone for late-onset forms of GM2 gangliosidosis;Maegawa;J. Biol. Chem.,2007

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