Enhancing delivery of nucleic acid therapeutics (NATs)

• Main Author: Ong, Kai Hong
• Other Authors: Phan Anh Tuan
• Format: Final Year Project
• Language: English
• Published: Nanyang Technological University 2021
• Subjects: Science::Biological sciences
• Online Access: https://hdl.handle.net/10356/148479
• Institution: Nanyang Technological University

Nucleic acid therapeutics (NATs) have the potential to be a treatment strategy for various diseases, by modulating gene expression. Its advancement over the past decade has resulted in several FDA approvals, with many more currently undergoing clinical trials. However, delivery of NATs to the site of interest continues to be a key challenge. In this study, we aim to exploit a specific cell surface receptor for enhancement of cellular delivery of NATs through receptor mediated endocytosis. To achieve this, we investigated the biochemistry of the ligand-receptor binding. We also established cell line models to over-express the receptor, and analysed improvement in activity of ligand conjugated NATs on these cells. Together, the results showed the potential of enhancing delivery of NATs using ligand-receptor interactions.