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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...
Autores principales: | Piechnik, Matthew, Sawamoto, Kazuki, Ohnishi, Hidenori, Kawamoto, Norio, Ago, Yasuhiko, Tomatsu, Shunji |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7279460/ https://www.ncbi.nlm.nih.gov/pubmed/32414007 http://dx.doi.org/10.3390/ijms21103433 |
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