A screening platform for lead therapeutic RNA oligonucleotides against haploinsufficiency diseases

Senior researcher

Aarhus University

Søren Lykke-Andersen, senior researcher, Department of Molecular Biology and Genetics, Aarhus University, has been awarded a Lundbeck Foundation LF Experiments research grant worth DKK 1,999,800.

This project proposes an exhaustive screening procedure that will allow the selection of lead compounds for future therapeutic interventions aiming to treat diseases caused by haploinsufficiency. The suggested approach ultimately relies on modified RNA oligonucleotides and can in principle be tailored to any haploinsufficiency, which may pave the way for a novel therapeutic paradigm to provide life-improving treatments to a broad spectrum of patients.