Veni grant for ENCORE member Annelot van Esbroeck
Annelot van Esbroeck receives an NWO Veni grant for research into personalized RNA therapy for rare genetic disorders. With her project Tailored, Targeted, Tolerable: A pipeline for personalized antisense oligonucleotides discovery she wants to make the development of tailored therapies faster and more widely applicable.
Annelot is a researcher within the Department of Clinical Genetics at Erasmus MC and a member of ENCORE. Her research aligns closely with ENCORE's ambition to better understand the genetic and molecular causes of developmental neurological disorders and translate them into targeted treatments.
A genetic change can have serious consequences, for example in the form of difficult-to-treat epilepsy. In some cases, antisense oligonucleotides (ASOs), a form of RNA therapy, can very specifically disable the harmful copy of a gene. However, the personalized development of such therapies is currently costly and time-consuming.
With her Veni research, Annelot aims to develop a more efficient approach. By using common genetic variations (SNPs) and existing stem cell models, she wants to accelerate the development and screening of personalized RNA therapy.