National Engineering Technology Research Center for Combined Vaccines, Wuhan 430207, China.
Wuhan Institute of Biological Products Co., Ltd., Wuhan 430207, China.
Int J Mol Sci. 2023 Apr 23;24(9):7736. doi: 10.3390/ijms24097736.
Gene therapy is a technique involving the modification of an individual's genes for treating a particular disease. The key to effective gene therapy is an efficient carrier delivery system. Viral vectors that have been artificially modified to lose their pathogenicity are used widely as a delivery system, with the key advantages of their natural high transduction efficiency and stable expression. With decades of development, viral vector-based gene therapies have achieved promising clinical outcomes. Currently, the three key vector strategies are based on adeno-associated viruses, adenoviruses, and lentiviruses. However, certain challenges, such as immunotoxicity and "off-target", continue to exist. In the present review, the above three viral vectors are discussed along with their respective therapeutic applications. In addition, the major translational challenges encountered in viral vector-based gene therapies are summarized, and the possible strategies to address these challenges are also discussed.
基因治疗是一种涉及个体基因修饰以治疗特定疾病的技术。有效的基因治疗的关键是高效的载体传递系统。经过人工修饰失去致病性的病毒载体被广泛用作传递系统，其主要优点是天然的高转导效率和稳定表达。经过几十年的发展，基于病毒载体的基因治疗已经取得了有希望的临床结果。目前，三种关键的载体策略基于腺相关病毒、腺病毒和慢病毒。然而，仍存在某些挑战，如免疫毒性和“脱靶”。在本综述中，讨论了上述三种病毒载体及其各自的治疗应用。此外，还总结了基于病毒载体的基因治疗中遇到的主要转化挑战，并讨论了解决这些挑战的可能策略。
