Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation

Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation

Gene replacement therapy is a rational therapeutic strategy and clinical intervention for neurodegenerative disorders like Canavan disease, a leukodystrophy caused by biallelic mutations in the aspartoacylase (ASPA) gene. We aimed to investigate whether simultaneous intravenous (i.v.) and intracereb...

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Bibliographic Details
Main Authors: Manuela Corti, Barry J. Byrne, Dominic J. Gessler, Grace Thompson, Samantha Norman, Jenna Lammers, Kirsten E. Coleman, Cristina Liberati, Melissa E. Elder, Maria L. Escolar, Ibrahim S. Tuna, Clementina Mesaros, Gary I. Kleiner, Deborah S. Barbouth, Heather L. Gray-Edwards, Nathalie Clement, Brian D. Cleaver, Guangping Gao
Format: Article
Language:English
Published: Elsevier 2023-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Canavan disease
AAV
immune modulation
dual route of administration
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050123000918

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http://www.sciencedirect.com/science/article/pii/S2329050123000918

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