Reduced Immunogenicity of Intraparenchymal Delivery of Adeno-Associated Virus Serotype 2 Vectors: Brief Overview

Wuh Liang Hwu, Shin Ichi Muramatsu, Bruria Gidoni-Ben-zeev

Research output: Contribution to journalArticlepeer-review

Abstract

Pre existing immunity to adeno-associated virus (AAV) poses a concern in AAV vec-tor–mediated gene therapy. Localized administration of low doses of carefully chosen AAV sero-types can mitigate the risk of an immune response. This article will illustrate the low risk of immune response to AAV serotype 2 vector–mediated gene therapy to the brain with support from clinical trial data in aromatic L-amino acid decarboxylase deficiency and Parkinson disease.

Original languageEnglish
Pages (from-to)185-190
Number of pages6
JournalCurrent Gene Therapy
Volume22
Issue number3
DOIs
StatePublished - Jun 2022

Keywords

  • Adeno-associated virus
  • aromatic L-amino acid decarboxylase deficiency
  • eladocagene exuparvovec
  • immunogenicity
  • rare disease
  • vectors

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