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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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Bibliografske podrobnosti
izdano v:Int J Mol Sci
Main Authors: Piechnik, Matthew, Sawamoto, Kazuki, Ohnishi, Hidenori, Kawamoto, Norio, Ago, Yasuhiko, Tomatsu, Shunji
Format: Artigo
Jezik:Inglês
Izdano: MDPI 2020
Teme:
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC7279460/
https://ncbi.nlm.nih.gov/pubmed/32414007
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms21103433
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