Paoletti Luca, De Clerck Kaat, De Coster Emma, Matricardi Pietro, Remaut Katrien, Peynshaert Karen
Department of Drug Chemistry and Technologies, Sapienza University of Rome, P.le A. Moro 5, 00185, Rome, Italy.
Lab of General Biochemistry and Physical Pharmacy, Faculty of Pharmaceutical Sciences, Ghent University, Ottergemsesteenweg 460, Ghent, 9000, Belgium.
Drug Deliv Transl Res. 2025 Jul 16. doi: 10.1007/s13346-025-01921-w.
Millions of people worldwide suffer from vision impairments, significantly affecting their quality of life and mental health and imposing a massive economic burden on healthcare. The eye, converting light into signals for the brain, plays a key role in vision, with the retina being especially vital yet susceptible to damage. Gene therapy holds promise for effectively treating vision-related diseases. However, in basic research, screening for effective gene carriers often lacks quantitative evaluation. One major challenge in translating promising nanoparticles from in vitro studies to (pre)clinical applications is the presence of retinal drug delivery barriers. Ex vivo experimentation could help overcome that by providing insights into these barriers early on, yet much of the current research relies heavily on qualitative assessments. Specifically, the lack of quantitative methods to assess nanoparticle penetration in the retina moved us to develop a flow cytometry-based method for large animal retinal tissue. Our method proved to efficiently quantify both nanoparticle uptake and transfection in the retina, corroborating our qualitative outcomes and positioning itself as a useful tool to support the drug and gene delivery community.
全球数百万人患有视力障碍，这严重影响了他们的生活质量和心理健康，并给医疗保健带来了巨大的经济负担。眼睛将光转化为大脑的信号，在视觉中起着关键作用，其中视网膜尤为重要，但也容易受到损伤。基因治疗有望有效治疗与视力相关的疾病。然而，在基础研究中，筛选有效的基因载体往往缺乏定量评估。将有前景的纳米颗粒从体外研究转化为（前）临床应用的一个主要挑战是存在视网膜药物递送障碍。体外实验可以通过尽早深入了解这些障碍来帮助克服这一问题，但目前的许多研究严重依赖定性评估。具体而言，缺乏评估纳米颗粒在视网膜中渗透的定量方法促使我们开发一种基于流式细胞术的大型动物视网膜组织方法。我们的方法证明能够有效地量化纳米颗粒在视网膜中的摄取和转染，证实了我们的定性结果，并成为支持药物和基因递送领域的有用工具。
