Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors

Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors

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Gene therapy using neurotropic adeno-associated virus vectors represents an emerging solution for genetic disorders affecting the central nervous system. The first approved central nervous system-targeting adeno-associated virus gene therapy, Zolgensma®, for treating spinal muscular atrophy is admin...

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Bibliographic Details
Main Authors: Makoto Horiuchi, Christian J. Hinderer, Jenny A. Greig, Cecilia Dyer, Elizabeth L. Buza, Peter Bell, Jessica A. Chichester, Peter M. Hayashi, Hanying Yan, Tamara Goode, James M. Wilson
Format: Article
Language:English
Published: Elsevier 2022-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
AAV
intrathecal
intravenous immunoglobulin
neutralizing antibodies
central nervous system
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050122001425

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