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

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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Bibliographic Details
Main Authors: Petr O. Ilyinskii, Alicia M. Michaud, Gina L. Rizzo, Christopher J. Roy, Sheldon S. Leung, Stephanie L. Elkins, Teresa Capela, Aparajita Chowdhury, Lina Li, Randy J. Chandler, Irini Manoli, Eva Andres-Mateos, Lloyd P.M. Johnston, Luk H. Vandenberghe, Charles P. Venditti, Takashi Kei Kishimoto
Format: Article
Language:English
Published: Elsevier 2021-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
ImmTOR rapamycin-encapsulated nanoparticles
gene therapy
immunogenicity mitigation
re-dosing
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121001170

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http://www.sciencedirect.com/science/article/pii/S2329050121001170

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