重组腺相关病毒:很有潜力的基因治疗载体

rAAV:a Promising Vehicle for Gene Therapy

腺相关病毒(AAV)是细小病毒家族的一员,为无包膜的线性单链DNA病毒.由于AAV具有长期潜伏于人体而不具有任何明显致病性等优点,人们对AAV作为一种理想的基因治疗载体给予了很大期望.但是,近来发现,这类载体在应用上有许多明显的缺陷,包括某些细胞膜上病毒受体数量极少,重组AAV载体位点特异性整合不足,AAV衣壳成分和转基因产物引起宿主的免疫反应等等.这些缺陷促使人们加大对AAV生物学特性和转染过程的研究,从而更好地对AAV载体进行改进,使新一代重组AAV载体具备基因治疗所必需的安全性、高效性和靶向性,以期更广泛地应用于临床.

Adeno-associated virus （AAV） is a member of the parvovirus family, single-stranded DNA-containing nonenveloped icosahedral viruses. AAVs have been regarded as promising vectors for human gene therapy as they have the capacity to establish long-term latency within human cells without any apparent pathogenicity. However, a lot of obvious defects in their applications have been revealed recently, including the paucity of cell surface receptors on some cells, the lack of site-specific integration by recombinant AAV vectors, and the host immune responses to AAV capsid components and transgene products and so forth. Driven by these defects, increasing efforts are being made to study biological properties and infectious pathway of AAVs. It is consequently optimized by the modification of the AAV vectors to produce new generation of recombinant AAV vectors with more security, efficiency and site-specific targeting, allowing AAVs to move forward into broader clinical application.