Oligonucleotide-based therapies, which involve the use of short DNA or RNA molecules to target specific genes, are offering highly targeted treatment options. However, conventional preclinical models often fall short in predicting their effectiveness in humans. To meet the need for more predictive data, researchers are turning to advanced human-relevant in vitro systems that allow for detailed analysis of oligonucleotide delivery, uptake, and gene knockdown in liver tissues.
Download the application note to learn:
- How galactosamine-conjugated oligonucleotides achieve greater gene knockdown than non-conjugated counterparts
- How liver microphysiological systems (MPS) enable dose-dependent analysis and repeat dosing strategies that reflect clinical treatment plans
- What makes MPS a powerful alternative to non-human primate studies for RNA therapeutic development