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

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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Bibliographic Details
Main Authors: Andreucci, A. (Author), Bulychev, A. (Author), Dale, E. (Author), Hu, X.S (Author), Iwamoto, N. (Author), Lamore, S. (Author), Lin, X. (Author), Liu, F. (Author), Liu, Y. (Author), Mohapatra, S. (Author), Patil, S. (Author), Purcell-Estabrook, E. (Author), Taborn, K. (Author), Vargeese, C. (Author), Yang, H. (Author), Yin, Y. (Author)
Format: Article
Language:English
Published: Cell Press 2022
Subjects:
amyotrophic lateral sclerosis
antisense oligonucleotide
C9orf72
dipeptide-repeat proteins
frontotemporal dementia
guanidine-containing backbone
mouse model
MT: Oligonucleotides: Therapies and Applications
poly-GP
pre-clinical study
stereopure
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