Department of Thoracic Surgery, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200025, China.
Biomolecules. 2024 Jul 25;14(8):904. doi: 10.3390/biom14080904.
Gene delivery therapy has emerged as a popular approach for the treatment of various diseases. However, it still poses the challenges of accumulation in target sites and reducing off-target effects. Aerosol gene delivery for the treatment of pulmonary diseases has the advantages of high lung accumulation, specific targeting and fewer systemic side effects. However, the key challenge is selecting the appropriate formulation for aerosol gene delivery that can overcome physiological barriers. There are numerous existing gene carriers under study, including viral vectors and non-viral vectors. With the development of biomaterials, more biocompatible substances have applied gene delivery via inhalation. Furthermore, many types of genes can be delivered through aerosol inhalation, such as DNA, mRNA, siRNA and CRISPR/Cas9. Aerosol delivery of different types of genes has proven to be efficient in the treatment of many diseases such as SARS-CoV-2, cystic fibrosis and lung cancer. In this paper, we provide a comprehensive review of the ongoing research on aerosol gene delivery therapy, including the basic respiratory system, different types of gene carriers, different types of carried genes and clinical applications.
基因传递治疗已成为治疗各种疾病的一种流行方法。然而,它仍然面临着在靶部位积累和减少脱靶效应的挑战。用于治疗肺部疾病的气溶胶基因传递具有高肺部积累、特异性靶向和较少的全身副作用的优点。然而,关键的挑战是选择适当的气溶胶基因传递制剂,以克服生理障碍。目前有许多正在研究的基因载体,包括病毒载体和非病毒载体。随着生物材料的发展,更多的生物相容性物质通过吸入进行基因传递。此外,许多类型的基因可以通过气溶胶吸入传递,如 DNA、mRNA、siRNA 和 CRISPR/Cas9。不同类型的基因的气溶胶传递已被证明在治疗许多疾病如 SARS-CoV-2、囊性纤维化和肺癌方面是有效的。本文对气溶胶基因传递治疗的研究进展进行了全面综述,包括基本呼吸系统、不同类型的基因载体、不同类型的携带基因和临床应用。