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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Detalles Bibliográficos
Autores principales: Horiuchi, Makoto, Hinderer, Christian J., Greig, Jenny A., Dyer, Cecilia, Buza, Elizabeth L., Bell, Peter, Chichester, Jessica A., Hayashi, Peter M., Yan, Hanying, Goode, Tamara, Wilson, James M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
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Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9593247/
https://www.ncbi.nlm.nih.gov/pubmed/36320416
http://dx.doi.org/10.1016/j.omtm.2022.09.017
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