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

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 subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system. Adverse events should also be reported to PTC Therapeutics at pharmacovigilance@ptcbio.com.

Eladocagene exuparvovec (UpstazaTM) is a gene therapy indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of AADC deficiency with a severe phenotype, currently licensed in the European Member States, Great Britain, Iceland, Liechtenstein, Northern Ireland and Norway.

Registration conditions differ internationally; always consult local prescribing information and/or Summary of Product Characteristics before prescribing any product.

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