4.4 Article

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

期刊

CURRENT GENE THERAPY
卷 22, 期 3, 页码 185-190

出版社

BENTHAM SCIENCE PUBL LTD
DOI: 10.2174/1566523221666210922155413

关键词

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

资金

  1. PTC Therapeutics

向作者/读者索取更多资源

This article illustrates 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.
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 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.

作者

我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。

评论

主要评分

4.4
评分不足

次要评分

新颖性
-
重要性
-
科学严谨性
-
评价这篇论文

推荐

暂无数据
暂无数据