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 an investigational gene therapy product currently being developed by PTC Therapeutics for the treatment of patients with deficiency of a protein called AADC. Eladocagene exuparvovec does not currently have a marketing authorization for the treatment of AADC deficiency.

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MED-ALL-AADC-2200023 | March 2022
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