(194) The introduction of Ligand for Enhanced Nuclear Delivery of Antisense Oligonucleotide
Introduction: Antisense oligonucleotides (ASOs) are versatile molecules designed to modulate RNA transcripts (1). As the most representative example, Spinraza is widely recognized as an ASO therapeutic for spinal muscular atrophy (SMA) (2). However, Spinraza requires high-dose administration due to limited research on ASO delivery, leading to increased side effects and costs (3). This study proposes a novel strategy that enhances ASO delivery by combining steroid hormone receptor-mediated transport with advanced lipid nanoparticle (LNP) technology to improve therapeutic efficacy.
Learning Objectives:
Upon completion, participant will be able to understand the limitations of current ASO therapeutics.
Upon completion, participant will be able to explore a novel ASO delivery strategy.
