Early postnatal expression mitigates immune responses to Cas9 in the murine central nervous system

A barrier in the development of adeno-associated virus (AAV) gene therapy is the immunogenicity of the AAV particles, and in some cases, the expressed transgene. The immunogenic risk is heightened when exogenous proteins, such as prokaryotic Cas9 nucleases, are used in gene editing. We documented th...

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Bibliographic Details
Main Authors: Robert Duba-Kiss, David R. Hampson
Format: Article
Language:English
Published: Elsevier 2025-09-01
Series:Molecular Therapy: Methods & Clinical Development
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Online Access:http://www.sciencedirect.com/science/article/pii/S2329050125001317
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