Preclinical evaluation of WVE-004, an investigational stereopure oligonucleotide for the treatment of C9orf72-associated ALS or FTD
Yuanjing Liu, Amy Andreucci, Naoki Iwamoto, Yuan Yin, Hailin Yang, Fangjun Liu, Alexey Bulychev, Xiao Hu, Xuena Lin, Sarah D. Lamore, Saurabh Patil, Susovan Mohapatra, Erin Purcell-Estabrook, Kristin Taborn, Elena Dale, Chandra Vargeese
Abstract
associated ALS or FTD.
Topics & Concepts
C9orf72Investigational DrugsMedicineOncologyFrontotemporal dementiaInternal medicineComputational biologyBiologyClinical trialDiseaseDementiaAmyotrophic Lateral Sclerosis ResearchNeurogenetic and Muscular Disorders Researchbiodegradable polymer synthesis and properties