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

被引：4

作者：

Hwu, Wuh-Liang ^{[1
]}

Muramatsu, Shin-Ichi ^{[2
,3
]}

Gidoni-Ben-Zeev, Bruria ^{[4
]}

机构：

[1] Natl Taiwan Univ Hosp, Dept Med Genet & Pediat, 7 Chung Shan S Rd, Taipei, Taiwan

[2] Jichi Med Univ, Ctr Open Innovat, Div Neurol Gene Therapy, 3311-1 Yakushiji, Shimotsuke, Tochigi 3290498, Japan

[3] Univ Tokyo, Ctr Gene & Cell Therapy, Inst Med Sci, Minato Ku, 4-6-1 Shirokanedai, Tokyo 1080071, Japan

[4] Tel Aviv Univ, Sackler Sch Med, Dept Pediat Neurol, IL-6997801 Tel Aviv, Israel

来源：

CURRENT GENE THERAPY | 2022年 / 22卷 / 03期

关键词：

Adeno-associated virus; aromatic L-amino acid decarboxylase deficiency; eladocagene exuparvovec; immunogenicity; vectors; rare disease; AMINO-ACID DECARBOXYLASE; CENTRAL-NERVOUS-SYSTEM; INNATE IMMUNE-RESPONSES; MEDIATED GENE-TRANSFER; OPEN-LABEL; PHASE-I; THERAPY; TRANSDUCTION; LIVER; AAV9;

D O I：

10.2174/1566523221666210922155413

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

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.

引用

页码：185 / 190

页数：6

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