Balakrishnan Balaji, Jayandharan Giridhara R
Department of Hematology & Centre for Stem Cell Research, Christian Medical College, Vellore-632004, Tamil Nadu, India.
Curr Gene Ther. 2014;14(2):86-100. doi: 10.2174/1566523214666140302193709.
Adeno-associated virus (AAV) based vectors have emerged as important tools for gene therapy in humans. The recent successes seen in Phase I/II clinical trials have also highlighted the issues related to the host and vector-related immune response that preclude the universal application of this promising vector system. A fundamental insight into the biological mechanisms by which AAV infects the host cell and a thorough understanding of the immediate and long-lived cellular responses to AAV infection is likely to offer clues and help design better intervention strategies to improve the therapeutic efficiency of AAV vectors. This article reviews the biology of AAV-host cellular interactions and outlines their application in the development of novel and improved AAV vector systems.
基于腺相关病毒（AAV）的载体已成为人类基因治疗的重要工具。在I/II期临床试验中取得的近期成功也凸显了与宿主和载体相关免疫反应有关的问题，这些问题妨碍了这种有前景的载体系统的广泛应用。深入了解AAV感染宿主细胞的生物学机制以及全面理解细胞对AAV感染的即时和长期反应，可能会提供线索并有助于设计更好的干预策略，以提高AAV载体的治疗效率。本文综述了AAV与宿主细胞相互作用的生物学，并概述了它们在新型和改良AAV载体系统开发中的应用。
