2021Current Gene TherapyOpen access

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

Shin‐ichi Muramatsu, Wuh‐Liang Hwu, Bruria Gidoni-Ben-Zeev

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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.

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What this paper is about

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.

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Available 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.

Key concepts: Adeno-associated virus, Immunogenicity, Genetic enhancement, Vector (molecular biology), Virology, Immune system, Serotype, Gene delivery

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Reduced Immunogenicity of Intraparenchymal Delivery of Adeno-Associated Virus Serotype 2 Vectors: Brief Overview — Research Paper | ScholarLens