Enhancement of liver-directed transgene expression at initial and repeat doses of AAV vectors admixed with ImmTOR nanoparticles

Systemic AAV (adeno-associated virus) gene therapy is a promising approach for the treatment of inborn errors of metabolism, but questions remain regarding its potency and durability. Tolerogenic ImmTOR nanoparticles encapsulating rapamycin have been shown to block the formation of neutralizing anti...

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Detalles Bibliográficos
Autores principales: Ilyinskii, Petr O., Michaud, Alicia M., Roy, Christopher J., Rizzo, Gina L., Elkins, Stephanie L., Capela, Teresa, Chowdhury, Aparajita C., Leung, Sheldon S., Kishimoto, Takashi K.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2021
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7904260/
https://www.ncbi.nlm.nih.gov/pubmed/33627416
http://dx.doi.org/10.1126/sciadv.abd0321

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