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AAV基因治疗产品的免疫反应评估

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随着生命科学技术的进步,基因治疗因其特异性和精准性等优势已成为新药研发的重点领域.其中,腺相关病毒(adeno-associated virus,AAV)载体是一种应用前景广阔的体内基因治疗载体,也是基因治疗产品载体研究的热点.目前,全球范围内已有7款AAV相关的基因治疗产品获批上市,并有多项临床试验正在进行.AAV载体的免疫原性较低,但部分人群体内存在针对AAV的预存免疫,其进入体内后也会诱导机体产生先天免疫反应,随后可能触发针对AAV衣壳和转基因产物的适应性免疫反应,从而影响药物的有效性和安全性.本综述旨在概述AAV基因治疗引起的免疫反应特征,并简要总结在临床前和临床研究中对AAV基因治疗免疫反应的评估,以期为AAV基因治疗药物评价提供参考.
Evaluation of Immune Response to AAV-Based Gene Therapy Products
With the advancement of life science technologies,gene therapy has emerged as a focal point in new drug development due to its specificity and precision.Among various vectors,adeno-associated virus(AAV)has gained prominence as an intracellular gene therapy vector,standing out as a hot research focus for gene therapy products.Currently,seven AAV-based gene therapy products have received regulatory approval and entered the market worldwide,and several clinical trials are underway.AAV vectors exhibit low immu-nogenicity,however,pre-existing immunity against AAV in certain populations may induce innate immune responses upon vector admin-istration.Subsequently,this may trigger adaptive immune reactions against the AAV capsid and transgene products,potentially influenc-ing the efficacy and safety of the therapeutic intervention.This review aims to outline the characteristics of immune responses induced by AAV-based gene therapy and briefly summarize the evaluation of immune response to AAV-based gene therapy in preclinical and clinical studies.The objective is to provide a reference for the evaluation of AAV-based gene therapy products.

Adeno-associated virus vectorAAVGene therapyEvaluation of immune response

何丹、夏艳、吴小兵

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北京锦篮基因科技有限公司,北京 100176

腺相关病毒载体 AAV 基因治疗 免疫反应评估

2024

中国医药导刊
国家食品药品监督管理局信息中心

中国医药导刊

影响因子:1.408
ISSN:1009-0959
年,卷(期):2024.26(3)
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