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Sex Difference Leads to Differential Gene Expression Patterns and Therapeutic Efficacy in Mucopolysaccharidosis IVA Murine Model Receiving AAV8 Gene Therapy

Adeno-associated virus (AAV) vector-based therapies can effectively correct some disease pathology in murine models with mucopolysaccharidoses. However, immunogenicity can limit therapeutic effect as immune responses target capsid proteins, transduced cells, and gene therapy products, ultimately res...

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Detalles Bibliográficos
Autores principales:	Piechnik, Matthew, Amendum, Paige C., Sawamoto, Kazuki, Stapleton, Molly, Khan, Shaukat, Fnu, Nidhi, Álvarez, Victor, Pachon, Angelica Maria Herreño, Danos, Olivier, Bruder, Joseph T., Karumuthil-Melethil, Subha, Tomatsu, Shunji
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2022
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9604118/ https://www.ncbi.nlm.nih.gov/pubmed/36293546 http://dx.doi.org/10.3390/ijms232012693

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