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

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...

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Detalhes bibliográficos
Main Authors: Yuanjing Liu, Amy Andreucci, Naoki Iwamoto, Yuan Yin, Hailin Yang, Fangjun Liu, Alexey Bulychev, Xiao Shelley Hu, Xuena Lin, Sarah Lamore, Saurabh Patil, Susovan Mohapatra, Erin Purcell-Estabrook, Kristin Taborn, Elena Dale, Chandra Vargeese
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2022-06-01
Colecção:Molecular Therapy: Nucleic Acids
Assuntos:
MT: Oligonucleotides: Therapies and Applications
antisense oligonucleotide
stereopure
amyotrophic lateral sclerosis
frontotemporal dementia
C9orf72
Acesso em linha:http://www.sciencedirect.com/science/article/pii/S216225312200083X
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