Preclinical evaluation of WVE-004, aninvestigational stereopure oligonucleotide forthe treatment of C9orf72-associated ALS or FTD

• Main Authors: Yuanjing Liu (Author), Amy Andreucci (Author), Naoki Iwamoto (Author), Yuan Yin (Author), Hailin Yang (Author), Fangjun Liu (Author), Alexey Bulychev (Author), Xiao Shelley Hu (Author), Xuena Lin (Author), Sarah Lamore (Author), Saurabh Patil (Author), Susovan Mohapatra (Author), Erin Purcell-Estabrook (Author), Kristin Taborn (Author), Elena Dale (Author), Chandra Vargeese (Author)
• Format: Book
• Published: Elsevier, 2022-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

A large hexanucleotide (G4C2) repeat expansion in the first intronic region of C9orf72 is the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Several mechanisms have been proposed to explain how the repeat expansion drives disease, and we hypothesi...