纳米技术照亮眼病基因治疗之路：从机遇到应用。
Nanotechnology Lighting the Way for Gene Therapy in Ophthalmopathy: From Opportunities toward Applications.
机构信息
Sichuan Provincial Key Laboratory for Human Disease Gene Study and Department of Laboratory Medicine, Sichuan Provincial People's Hospital, University of Electronic Science and Technology of China, Chengdu 610072, China.
Health Management Center, Sichuan Provincial People's Hospital, University of Electronic Science and Technology of China, Chengdu 610072, China.
出版信息
Molecules. 2023 Apr 15;28(8):3500. doi: 10.3390/molecules28083500.
Hereditary ophthalmopathy is a well-described threat to human visual health affecting millions of people. Gene therapy for ophthalmopathy has received widespread attention with the increasing understanding of pathogenic genes. Effective and safe delivery of accurate nucleic acid drugs (NADs) is the core of gene therapy. Efficient nanodelivery and nanomodification technologies, appropriate targeted genes, and the choice of drug injection methods are the guiding lights of gene therapy. Compared with traditional drugs, NADs can specifically change the expression of specific genes or restore the normal function of mutant genes. Nanodelivery carriers can improve targeting and nanomodification can improve the stability of NADs. Therefore, NADs, which can fundamentally solve pathogeny, hold great promise in the treatment of ophthalmopathy. This paper reviews the limitations of ocular disease treatment, discusses the classification of NADs in ophthalmology, reveals the delivery strategies of NADs to improve bioavailability, targeting, and stability, and summarizes the mechanisms of NADs in ophthalmopathy.
遗传性眼病是一种严重威胁人类视觉健康的疾病，影响着数百万人。随着对致病基因认识的不断深入，眼病的基因治疗受到了广泛关注。将准确的核酸药物（NADs）有效且安全地递送至靶部位是基因治疗的核心。高效的纳米递药和纳米修饰技术、合适的靶向基因以及药物注射方法的选择是基因治疗的指导原则。与传统药物相比，NADs 可以特异性地改变特定基因的表达或恢复突变基因的正常功能。纳米递药载体可以提高靶向性，纳米修饰可以提高 NADs 的稳定性。因此，从根本上解决病因的 NADs 在眼病治疗中具有广阔的应用前景。本文综述了眼部疾病治疗的局限性，讨论了 NADs 在眼科中的分类，揭示了提高 NADs 生物利用度、靶向性和稳定性的递药策略，并总结了 NADs 在眼病中的作用机制。
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