Using Antisense Oligonucleotides to Increase mRNA Translation

Antisense technologies are emerging as an exciting therapeutic approach for neurodegenerative diseases, with the development of Spinraza as a first-in-class antisense oligonucleotide (ASO) therapy for Spinal Muscular Atrophy and several late-stage ASO clinical trials in the works for ALS. This project will develop a new ASO approach to upregulate protein expression for disease mutations affecting FTD and ALS. This team will use both targeted and screening approaches to discover ASOs that can lead to increased protein synthesis at the level of translation.