Gene therapy vectors based on adeno-associated virus: characteristics and applications to acquired and inherited diseases (review).
Gene therapy vectors based on adeno-associated virus: characteristics and applications to acquired and inherited diseases (review).
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DOI：
10.3892/ijmm.6.4.363
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发表时间：
2000-10
影响因子：
5.4
通讯作者：
T. Athanasopoulos;S. Fabb;G. Dickson
中科院分区：
文献类型：
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作者：
T. Athanasopoulos;S. Fabb;G. Dickson
腺相关病毒（Adeno-associated virus，AAV）是一种缺陷型细小病毒，30多年前被发现。该病毒的非致病性、广泛的嗜性和感染性、位点特异性整合和长期持久性是其在人类基因治疗中应用的主要研究方向。rAAV研究领域已经取得了相当大的进展：已经实现了1014 p/ml的滴度，设计用于产生无辅助病毒的质粒系统，结合了rAAV ITR的性质和来自Ad/HS载体的大序列容量的嵌合载体，以及基于形成多联体的异源二聚化的革命性内含子策略，已经扩大了载体容量。肌肉细胞和神经元（有丝分裂后细胞）是rAAV递送的最有效靶标之一，并且已经鉴定了AAV受体和共受体。本综述将描述rAAV技术领域的进展，克服了载体作为基因递送系统的某些局限性，并概述了涉及这些重组载体用于治疗获得性和遗传性疾病的应用。
Adeno-associated virus (AAV), a defective parvovirus, was discovered more than 30 years ago. Interest in this virus for human gene therapy applications focuses on its non-pathogenicity, broad tropism and infectivity, site-specific integration and long-term persistence. The field of rAAV research has considerably advanced: titers of 1014 p/ml have been achieved, plasmid systems devised to produce helper-free viruses, chimaeric vectors combining properties of rAAV ITRs and large sequence capacity from Ad/HS vectors in parallel with the revolutionary intron strategy based on heterodimerisation of the forming concatamers have expanded the vector capacity. Muscle cells and neurons (post-mitotic cells) are amongst the most efficient targets of rAAV delivery and AAV receptors and co-receptors have been identified. This review will describe advances in the field of rAAV technology that overcome certain limitations of the vector as a gene delivery system and overview applications involving these recombinant vectors for the treatment of acquired and inherited diseases.