Next-generation anti-sense molecules for Parkinson's disease therapy.
A nationally funded collaborative research project aiming at the development of next generation anti-sense molecules for Parkinson's disease therapy. The ASO-based study on Parkinson's disease (PD), conducted in humanized rats and iPSC neurons from patients with PD, yielded significant findings.
Reduced monomeric and aggregated alpha-synuclein levels; increased locomotor activity and enhanced olfactory function — without side effects or neurotoxicity.
Restored cytokine expression to wild-type levels, suggesting an immunomodulatory effect.
Phosphoprotein multiplexing highlighted restoration of activated signaling pathways, underscoring ASO3's potential in reversing PD pathology.
Pharmacokinetic analysis showed ASO3 remained stable in rat brains for an extended period. Comprehensive bioinformatics analysis of RNAseq data helped identify molecular biomarkers of PD.
e-NIOS performed comprehensive bioinformatics analysis of RNAseq data from this study using the BioInfoMiner platform, helping assess mechanisms of action of ASO3 and identify molecular biomarkers of Parkinson's disease.