Department of Stomatology Tongji Hospital Tongji Medical College Huazhong University of Science and Technology Wuhan Hubei Province 430030 China.
Department of Oncology Tongji Hospital Tongji Medical College Huazhong University of Science and Technology Wuhan Hubei Province 430030 China.
Adv Sci (Weinh). 2021 May 16;8(14):2100540. doi: 10.1002/advs.202100540. eCollection 2021 Jul.
Gene therapy provides a promising strategy for curing monogenetic disorders and complex diseases. However, there are challenges associated with the use of viral delivery vectors. The advent of nanomedicine represents a quantum leap in the application of gene therapy. Recent advances in stimulus-responsive nonviral nanocarriers indicate that they are efficient delivery systems for loading and unloading of therapeutic nucleic acids. Some nanocarriers are responsive to cues derived from the internal environment, such as changes in pH, redox potential, enzyme activity, reactive oxygen species, adenosine triphosphate, and hypoxia. Others are responsive to external stimulations, including temperature gradients, light irradiation, ultrasonic energy, and magnetic field. Multiple stimuli-responsive strategies have also been investigated recently for experimental gene therapy.
基因治疗为治疗单基因疾病和复杂疾病提供了有前途的策略。然而,使用病毒传递载体存在一些挑战。纳米医学的出现代表了基因治疗应用的质的飞跃。最近刺激响应型非病毒纳米载体的进展表明,它们是有效装载和卸载治疗性核酸的递药系统。一些纳米载体对来源于内部环境的信号有响应,例如 pH 值、氧化还原电势、酶活性、活性氧物种、三磷酸腺苷和缺氧的变化。其他则对外部刺激有响应,包括温度梯度、光照射、超声能量和磁场。最近还研究了多种刺激响应策略用于实验性基因治疗。