E03 : ASCEND: A Novel Brain-Shuttle Antisense Therapy for Targeted RNA Repair in ALS


Students Taeyeon Eum
Eva Duan
School HDSB - Oakville Trafalgar High School - Oakville
Level Senior 11/12 - Grade 11
Group Group 9 - Health Sciences II
Abstract The purpose of ASCEND is to develop a novel brain-shuttle antisense therapy capable of crossing the blood-brain barrier to repaire TDP-43 induced RNA mis-splicing in ALS. Using STMN2 restoration as a proof of concept, this project aims to create a scalable, precision RNA repaire platform designed to restore neuronal function in neurodegenerative disease.