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健康人群血清 IgG 及中和因子针对腺相关病毒(AAV)血清型 1、2、5、6、8 和 9 的流行率:对使用 AAV 载体的基因治疗的影响。

Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors.

机构信息

Laboratoire d'Immunologie, Genethon R&D, Evry Cedex, France.

出版信息

Hum Gene Ther. 2010 Jun;21(6):704-12. doi: 10.1089/hum.2009.182.


DOI:10.1089/hum.2009.182
PMID:20095819
Abstract

Adeno-associated viruses (AAVs) are small, nonenveloped single-stranded DNA viruses that require helper viruses to facilitate efficient replication. Despite the presence of humoral responses to the wild-type AAV in humans, AAV remains one of the most promising candidates for therapeutic gene transfer to treat many genetic and acquired diseases. Characterization of the IgG subclass responses to AAV and study of the prevalence of both IgG and neutralizing factors to AAV types 1, 2, 5, 6, 8, and 9 in the human population are of importance for the development of new strategies to overcome these immune responses. Natural exposure to AAV types 1, 2, 5, 6, 8, and 9 can result in the production of antibodies from all four IgG subclasses, with a predominant IgG1 response and low IgG2, IgG3, and IgG4 responses. Prevalences of anti-AAV1 and -AAV2 total IgG determined by enzyme-linked immunosorbent assay were higher (67 and 72%) than those of anti-AAV5 (40%), anti-AAV6 (46%), anti-AAV8 (38%), and anti-AAV9 (47%). Furthermore, data showed that cross-reactions are important. The two highest neutralizing factor seroprevalences were observed for AAV2 (59%) and AAV1 (50.5%) and the lowest were observed for AAV8 (19%) and AAV5 (3.2%). Vectors based on AAV5, AAV8, and AAV9 may have an advantage for gene therapy in humans. Furthermore, among individuals seropositive for AAV5, AAV8, and AAV9, about 70-100% present low titers. Better characterization of the preexisting humoral responses to the AAV capsid and cross-reactivity will allow development of new strategies to circumvent AAV acquired immune responses.

摘要

腺相关病毒(AAV)是小型、无包膜的单链 DNA 病毒,需要辅助病毒来促进有效的复制。尽管人类对野生型 AAV 存在体液反应,但 AAV 仍然是治疗许多遗传和获得性疾病的基因转移最有前途的候选者之一。AAV 对 IgG 亚类反应的特征描述以及对人类人群中 AAV 类型 1、2、5、6、8 和 9 的 IgG 和中和因子的流行率的研究,对于开发克服这些免疫反应的新策略非常重要。自然暴露于 AAV 类型 1、2、5、6、8 和 9 可导致所有四个 IgG 亚类产生抗体,以 IgG1 反应为主,IgG2、IgG3 和 IgG4 反应较低。通过酶联免疫吸附试验确定的抗 AAV1 和 -AAV2 总 IgG 的流行率(分别为 67%和 72%)高于抗 AAV5(40%)、抗 AAV6(46%)、抗 AAV8(38%)和抗 AAV9(47%)。此外,数据表明交叉反应很重要。AAV2(59%)和 AAV1(50.5%)的中和因子血清阳性率最高,AAV8(19%)和 AAV5(3.2%)的中和因子血清阳性率最低。基于 AAV5、AAV8 和 AAV9 的载体可能在人类基因治疗中具有优势。此外,在 AAV5、AAV8 和 AAV9 血清阳性的个体中,约 70-100%的个体呈现低滴度。更好地描述对 AAV 衣壳的预先存在的体液反应和交叉反应性将允许开发新策略来规避 AAV 获得性免疫反应。

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Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors.

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