Huang Baihao, De Smedt Stefaan C, De Vos Winnok H, Braeckmans Kevin
Laboratory of General Biochemistry and Physical Pharmacy, Faculty of Pharmaceutical Sciences, Ghent University, Ghent, Belgium.
Laboratory of Cell Biology and Histology, Department of Veterinary Sciences, University of Antwerp, Antwerp, Belgium.
Drug Deliv. 2025 Dec;32(1):2502346. doi: 10.1080/10717544.2025.2502346. Epub 2025 May 14.
Gene therapy has evolved into a clinically viable strategy, with several approved products demonstrating its therapeutic potential for genetic disorders, cancer, and infectious diseases, and it has ample applications in regenerative medicine. Its success depends on the ability to efficiently and specifically deliver therapeutic nucleic acids (NAs) into target cells. Although viral or chemical carriers have been used in pioneering applications, safety concerns, and variable delivery efficiencies have prompted the search for alternative delivery vehicles. Light-mediated strategies have gained particular interest due to their biocompatibility and ability to improve the intracellular delivery efficiency. In this review, we focus on recent advancements in the development of light-triggered NA delivery carriers and discuss how they can be designed to overcome specific intracellular barriers. Additionally, we discuss notable therapeutic applications and highlight challenges and opportunities for translating this technology to a clinical setting.
基因治疗已发展成为一种具有临床可行性的策略，有几种获批产品展示了其在治疗遗传疾病、癌症和传染病方面的治疗潜力，并且在再生医学中有广泛应用。其成功取决于能否高效且特异性地将治疗性核酸（NAs）递送至靶细胞。尽管病毒或化学载体已用于开创性应用，但安全问题和可变的递送效率促使人们寻找替代递送载体。光介导策略因其生物相容性和提高细胞内递送效率的能力而备受关注。在本综述中，我们重点关注光触发核酸递送载体开发的最新进展，并讨论如何设计它们以克服特定的细胞内障碍。此外，我们还讨论了显著的治疗应用，并强调了将该技术转化为临床应用所面临的挑战和机遇。
