Recent Advances in the Analysis Full/Empty Capsid Ratio and Genome Integrity of Adeno-associated Virus (AAV) Gene Delivery Vectors

doi:10.2174/1566524020999200730181042

摘要

腺相关病毒(Adeno-associated virus, AAV)是最有前途的具有长期基因表达和疾病纠正功能的病毒基因载体之一，在人体临床试验中具有高效和卓越的安全性。在AAV载体的生产过程中，有几个质量控制(QC)参数需要严格监测，以符合临床安全性和有效性。本文简要介绍了最常用的AVV生产和纯化方法，重点介绍了用于测定全/空衣壳比和被包裹治疗性DNA产品完整性的分析技术。

关键词: 腺相关病毒(AAV)，良好生产规范，全/空衣壳比，电子显微镜，阴离子交换色谱，AAV载体基因组完整性，高通量测序。

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Current Molecular Medicine

Title:Recent Advances in the Analysis Full/Empty Capsid Ratio and Genome Integrity of Adeno-associated Virus (AAV) Gene Delivery Vectors

Volume: 20 Issue: 10

关键词: 腺相关病毒(AAV)，良好生产规范，全/空衣壳比，电子显微镜，阴离子交换色谱，AAV载体基因组完整性，高通量测序。

摘要: Adeno-associated virus (AAV) is one of the most promising viral gene delivery vectors with long-term gene expression and disease correction, featuring high efficiency and excellent safety in human clinical trials. During the production of AAV vectors, there are several quality control (QC) parameters that should be rigorously monitored to comply with clinical safety and efficacy. This review gives a short summary of the most frequently used AVV production and purification methods, focusing on the analytical techniques applied to determine the full/empty capsid ratio and the integrity of the encapsidated therapeutic DNA of the products.

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Recent Advances in the Analysis Full/Empty Capsid Ratio and Genome Integrity of Adeno-associated Virus (AAV) Gene Delivery Vectors, Current Molecular Medicine 2020; 20 (10) . https://dx.doi.org/10.2174/1566524020999200730181042

DOI https://dx.doi.org/10.2174/1566524020999200730181042	Print ISSN 1566-5240
Publisher Name Bentham Science Publisher	Online ISSN 1875-5666