ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia

A major barrier to adeno-associated virus (AAV) gene therapy is the inability to re-dose patients due to formation of vector-induced neutralizing antibodies (Nabs). Tolerogenic nanoparticles encapsulating rapamycin (ImmTOR) provide long-term and specific suppression of adaptive immune responses, all...

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Detalles Bibliográficos
Autores principales: Ilyinskii, Petr O., Michaud, Alicia M., Rizzo, Gina L., Roy, Christopher J., Leung, Sheldon S., Elkins, Stephanie L., Capela, Teresa, Chowdhury, Aparajita, Li, Lina, Chandler, Randy J., Manoli, Irini, Andres-Mateos, Eva, Johnston, Lloyd P.M., Vandenberghe, Luk H., Venditti, Charles P., Kishimoto, Takashi Kei
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8399083/
https://www.ncbi.nlm.nih.gov/pubmed/34485611
http://dx.doi.org/10.1016/j.omtm.2021.06.015

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8399083/
https://www.ncbi.nlm.nih.gov/pubmed/34485611
http://dx.doi.org/10.1016/j.omtm.2021.06.015

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