靶向重组AAV基因治疗的备选衣壳策略

S. Loiler, T. Flotte
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引用次数: 1

摘要

腺相关病毒(AAV)新血清型的出现扩大了组织趋向性,并允许临床相关数量的细胞被rAAV载体转导。未来用AAV载体进行基因治疗的两个最大障碍是组织/靶细胞特异性和位点特异性整合。本期的其他评论将着眼于特定站点集成方面的进展。本文将重点介绍AAV载体的向性和组织/靶细胞特异性的最新进展。
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Alternative capsid strategies for targeting recombinant AAV gene therapy
The advent of new serotypes of adeno-associated viruses (AAV) has expanded the tissue tropism and allowed clinically relevant numbers of cells to be transduced by rAAV vectors. The two most significant obstacles in the way of future gene therapies with AAV vectors are tissue/target cell specificity and sitel-specific integration. Other reviews contained in this issue will look at advances in site-specific integration. This review will focus on the latest developments in AAV vector tropism and tissue/ target cell specificity.
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