Reduced Immunogenicity of Intraparenchymal Delivery of Adenoassociated Virus Serotype 2 Vectors: Brief Overview

(E-pub Ahead of Print)

Author(s): Wuh-Liang Hwu, Shin-Ichi Muramatsu*, Bruria Gidoni-Ben-Zeev

Journal Name: Current Gene Therapy


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Abstract:

Pre existing immunity to Adeno-Associated Virus (AAV) poses a concern in AAV vector– mediated gene therapy. Localized administration of low doses of carefully chosen AAV serotypes 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.

Keywords: Adeno-associated virus, aromatic L-amino acid decarboxylase deficiency, eladocagene exuparvovec, immunogenicity, vectors, rare disease.

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(E-pub Ahead of Print)
DOI: 10.2174/1566523221666210922155413

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