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
- PTC Therapeutics
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。
推荐
暂无数据