Article

Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptides in vivo

2022-09-23

Abstract excerpt

<title>Abstract</title> <p>Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that affects motor neurons, causing progressive muscle weakness and respiratory failure. The presence of an expanded hexanucleotide repeat in chromosome 9 open reading frame 72 (<italic>C9ORF72</italic>) accounts for most cases of familial ALS and frontotemporal dementia (FTD). To determine if suppressing expressio...

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Literature Corpus work: 7c352b57-a480-507f-a5f8-6920a583e97c
DOI: 10.21203/rs.3.rs-2081412/v1

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