Reduced Immunogenicity of Intraparenchymal Delivery of Adeno-Associated Virus Serotype 2 Vectors: Brief Overview
Shin‐ichi Muramatsu, Wuh‐Liang Hwu, Bruria Gidoni-Ben-Zeev
Abstract
Shin‐ichi Muramatsu, Wuh‐Liang Hwu, Bruria Gidoni-Ben-Zeev
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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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