Asokan Aravind
Gene Therapy Center, The University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Discov Med. 2010 May;9(48):399-403.
Adeno-associated viral (AAV) vectors have emerged in recent years as powerful tools for therapeutic gene transfer. Successes in clinical trials and the discovery of several hundreds of naturally occurring AAV isolates have triggered efforts to understand and manipulate this deceptively simple parvovirus for a myriad of gene therapy applications. Exciting breakthroughs based on directed evolution of novel tissue-specific variants from combinatorial AAV libraries have been reported. Recent approaches driven by the availability of structural information have yielded a new generation of reengineered AAV vectors.
近年来，腺相关病毒（AAV）载体已成为治疗性基因转移的强大工具。临床试验的成功以及数百种天然存在的AAV分离株的发现，引发了人们为多种基因治疗应用而理解和操纵这种看似简单的细小病毒的努力。基于从组合AAV文库中定向进化新型组织特异性变体的令人兴奋的突破已有报道。由结构信息的可用性驱动的最新方法已产生了新一代重新设计的AAV载体。
