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

A large hexanucleotide (G(4)C(2)) 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 hypot...

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Detalles Bibliográficos
Autores principales: Liu, Yuanjing, Andreucci, Amy, Iwamoto, Naoki, Yin, Yuan, Yang, Hailin, Liu, Fangjun, Bulychev, Alexey, Hu, Xiao Shelley, Lin, Xuena, Lamore, Sarah, Patil, Saurabh, Mohapatra, Susovan, Purcell-Estabrook, Erin, Taborn, Kristin, Dale, Elena, Vargeese, Chandra
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9092894/
https://www.ncbi.nlm.nih.gov/pubmed/35592494
http://dx.doi.org/10.1016/j.omtn.2022.04.007

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