Wouter van Rheenen and Vamshidhar R. Vangoor

Antisense therapies targeting ALS in induced neurons

ALS is a fatal neurodegenerative disease that can be caused by a range of rare genetic mutations. This project aims to develop gene-targeted therapies to silence newly discovered ALS-associated mutations. The team will establish a scalable pipeline for the generation and characterization of patient-derived motor neurons and use this platform to screen and optimize allele-specific antisense oligonucleotides. The project aims to nominate a lead candidate for safety testing and future first-in-human precision therapy.