DataVXACS NanoPaper
journal article·2025-10-14

Disulfide-Appended siRNA Nanoeyedrops: Noninvasive Gene Therapy for Targeting Retinal Angiogenesis

Shuya Liu, Dongliang Wang, Sishi Ye, Junjie Zhang, Beiyan Du, Zhijie Lian, Wen Yang, Jie Zhou, Qi Chen, Juan Li, Jingying Li, Huanghao Yang

ACS Nano· Volume 19· Issue 40
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Source abstract

Abstract

Given the significant advantages the eye presents as a target organ for gene therapy, it has remained at the forefront of translational research in this field. However, gene delivery to the desired tissue, particularly the retina, via a noninvasive route of administration poses a substantial physical challenge due to the presence of multiple ocular barriers. Here, we develop disulfide-appended small interfering RNA (DS-siRNA) Nanoeyedrops that target pathological retinal angiogenesis. These Nanoeyedrops enable noninvasive and effective gene delivery into the retina through the thiol-mediated uptake route. We have demonstrated that the thiol-mediated uptake route represents a powerful approach capable of overcoming ocular barriers. Ultimately, we have applied these Nanoeyedrops to deliver VEGF siRNA, which has exhibited substantial effects in both choroidal neovascularization and retinoblastoma mouse models. It serves as a highly efficient delivery system that enables noninvasive ocular gene delivery with high bioavailability, thereby offering a promising strategy for treating retinal diseases.
Source: openalex_inverted_index · Quality: baseline

Research context

Nanomaterials

OpenAlex topics: RNA Interference and Gene Delivery · Advanced biosensing and bioanalysis techniques · Nanoplatforms for cancer theranostics

SciVX discovery tags are terminology-based. OpenAlex topics are source-provided classifications.

Affiliations

New Cornerstone Science Laboratory, MOE Key Laboratory for Analytical Science of Food Safety and Biology, College of Chemistry

Fuzhou University

State Key Laboratory of Ophthalmology

Zhongshan Ophthalmic Center, SunYat-sen University

College of Biological Science and Engineering

Department of Bioengineering and Biopharmaceutics, School of Pharmacy

Fujian Medical University

College of medicine

Zhejiang Cancer Hospital, The Key Laboratory of Zhejiang Province for Aptamers and Theragnostic

Hangzhou Institute of Medicine (HIM), Chinese Academy of Sciences

Funding

National Key Research and Development Program of China · Natural Science Foundation of Fujian Province · National Natural Science Foundation of China · Outstanding Youth Foundation of Fujian Province · Zhejiang Leading Innovation and Entrepreneurship Team · Pioneer Research and Development Program of Zhejiang

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