Publication: Reduced immunogenicity of intraparenchymal delivery of adeno-associated virus serotype 2 vectors: Brief overview

This perspectives article, published in Current Gene Therapy, describes the low risk of immune response to adeno-associated virus serotype 2 (AAV2) vector-mediated gene therapy to the brain, with support from clinical trial data in aromatic L-amino acid decarboxylase (AADC) deficiency

Understand the role of pre-existing neutralizing antibodies and how they may impact the efficacy and safety of adeno-associated virus (AAV) vector-mediated gene therapy
Learn the differences between systemic and localized gene therapy and the features of different sites for gene therapy administration
Review clinical data from AAV2 vector-mediated gene therapy in AADC deficiency

Hwu WL, Muramatsu SI, Gidoni-Ben-Zeev B. Curr Gene Ther. 2021 Sep 22. Online ahead of print

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Eladocagene exuparvovec is currently an investigational gene therapy product in the US. Eladocagene exuparvovec has been granted marketing authorization by the European Commission for the European Union Member States, Iceland, Norway, Northern Ireland, and Lichtenstein and granted marketing authorization by the UK MHRA for Great Britain.

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Publication: Reduced immunogenicity of intraparenchymal delivery of adeno-associated virus serotype 2 vectors: Brief overview

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