Søren Lykke-Andersen

Treatment of haploinsufficiency disorders through targeted upregulation of protein expression using artificial small RNA

Applicant

Søren Lykke-Andersen

Project drscription

We aim to develop customizable nucleotide-based drugs for haploinsufficiency disorders, which are diseases caused by loss-of-function of one autosomal gene copy, resulting in a halving of the protein amount. There are ~350 verified haploinsufficiencies, including many severe ailments like cancer and neurodevelopmental disorders. With most lacking treatment options, these disorders represent a significant unmet medical need, and therapeutic interventions that increase functional protein levels are in high demand. Our approach involves targeting the mRNA with small artificial RNAs to boost protein levels from the healthy allele. In principle, the method can be designed for any haploinsufficiency, and the specific mutation is irrelevant.

Institution

Aarhus University