WVE-004, an investigational stereopure antisense oligonucleotide for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) (2302)
Yuanjing Liu, Amy Andreucci, Naoki Iwamoto, Yuan Yin, Hailin Yang, Fangjun Liu, Saurabh Patil, Susovan Mohapatra, Erin Purcell-Estabrook, Kristin Taborn, Elena Dale, Chandra Vargeese
Abstract
To determine the effects of WVE-004 on C9orf72 G4C2 repeat-containing transcripts, DPR proteins, and C9orf72 protein levels.
Topics & Concepts
Amyotrophic lateral sclerosisFrontotemporal dementiaMedicineC9orf72NeuroscienceDementiaPsychologyDiseaseInternal medicineAmyotrophic Lateral Sclerosis ResearchNeurogenetic and Muscular Disorders ResearchRNA Research and Splicing