Evading the AAV Immune Response in Mucopolysaccharidoses

The humoral immune response elicited by adeno-associated virus (AAV)-mediated gene therapy for the treatment of mucopolysaccharidoses (MPS) poses a significant challenge to achieving therapeutic levels of transgene expression. Antibodies targeting the AAV capsid as well as the transgene product dimi...

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Bibliographic Details
Main Authors: Matthew Piechnik, Kazuki Sawamoto, Hidenori Ohnishi, Norio Kawamoto, Yasuhiko Ago, Shunji Tomatsu
Format: Article
Language:English
Published: MDPI AG 2020-05-01
Series:International Journal of Molecular Sciences
Subjects:
Online Access:https://www.mdpi.com/1422-0067/21/10/3433