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Bound Protein and Peptide-Based Strategies for Adeno-associated Virus Vector-Mediated Gene Therapy: Where do We Stand Now?
Human Gene Therapy ( IF 3.9 ) Pub Date : 2020-11-12 , DOI: 10.1089/hum.2020.193
Xintao Zhang 1 , Zheng Chai 1 , R Jude Samulski 1, 2 , Chengwen Li 1, 3, 4
Affiliation  

Recombinant adeno-associated virus (rAAV) vectors have become one of the most promising and efficacious delivery vehicles for human gene therapy; however, low infectivity remains a major ongoing obstacle in the clinical application of rAAV vectors. Multiple strategies, including rAAV capsid modification and the application of pharmacological reagents, have been explored to enhance rAAV vector gene delivery. Recently, a new strategy using native proteins or various peptides has shown promise for increasing rAAV transduction locally or globally. This review summarizes the current status of protein- and peptide-based strategies and mechanisms to modulate rAAV transduction. We also provide a potential insight regarding the design of effective approaches for rAAV transduction enhancement in future clinical studies.

中文翻译:

基于结合蛋白和肽的腺相关病毒载体介导的基因治疗策略:我们现在处于什么位置?

重组腺相关病毒 (rAAV) 载体已成为人类基因治疗最有前途和最有效的载体之一;然而,低传染性仍然是 rAAV 载体临床应用的主要障碍。已经探索了多种策略,包括 rAAV 衣壳修饰和药理试剂的应用,以增强 rAAV 载体基因传递。最近,一种使用天然蛋白质或各种肽的新策略已显示出在局部或全球增加 rAAV 转导的前景。本综述总结了基于蛋白质和肽的调节 rAAV 转导的策略和机制的现状。我们还提供了有关在未来临床研究中设计增强 rAAV 转导的有效方法的潜在见解。
更新日期:2020-11-17
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