Home | Central nervous system | Aromatic L-amino acid decarboxylase (AADC) deficiency | Publications | Publication: Reduced immunogenicity of intraparenchymal delivery of adeno-associated virus serotype 2 vectors: Brief overview

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

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MED-ALL-PTC-AADC-2500006 | September 2025

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

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MED-ALL-AADC-2200023 | June 2025

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

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