Pre-clinical Gene Therapy with AAV9/AGA in Aspartylglucosaminuria Mice Provides Evidence for Clinical Translation-Reference-Cited by-同舟云学术

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Pre-clinical Gene Therapy with AAV9/AGA in Aspartylglucosaminuria Mice Provides Evidence for Clinical Translation

Published:2021-03 Issue:3 Volume:29 Page:989-1000
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Chen Xin,Snanoudj-Verber Sarah,Pollard Laura,Hu Yuhui,Cathey Sara S.,Tikkanen Ritva,Gray Steven J.

Funder

Rare Trait Hope Fund

UNC TraCS Institute

AFM-Téléthon

Bowles Center for Alcohol Studies

Lineberger Comprehensive Center

Carolina Institute for Developmental Disabilities

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference43 articles.

1. Aspartylglycosaminuria: a review;Arvio;Orphanet J. Rare Dis.,2016

2. Progressive nature of aspartylglucosaminuria;Arvio;Acta Paediatr.,2002

3. Bone marrow transplantation for aspartylglucosaminuria: follow-up study of transplanted and non-transplanted patients;Arvio;J. Pediatr.,2001

4. Enzyme replacement therapy in a mouse model of aspartylglycosaminuria;Dunder;FASEB J.,2000

5. Early initiation of enzyme replacement therapy improves metabolic correction in the brain tissue of aspartylglycosaminuria mice;Dunder;J. Inherit. Metab. Dis.,2010

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1. A 37-Year-Old Man With Intellectual Disability Discovered to Have Aspartylglucosaminuria;Neurology Genetics;2024-06

2. Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study;Molecular Therapy - Methods & Clinical Development;2024-03

3. Aspartylglucosaminuria;Genetic Syndromes;2023-12-20

4. Genome‐wide association and replication studies for handedness in a Korean community‐based cohort;Brain and Behavior;2023-06-20

5. A new horizon in the phosphorylated sites of AGA: the structural impact of C163S mutation in aspartylglucosaminuria through molecular dynamics simulation;Journal of Biomolecular Structure and Dynamics;2023-06-19

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