Article

Potent Mixed Backbone Antisense Oligonucleotide Safety Suppressed Expression of Mutant C9ORF72 Transcripts and Polypeptides: First in Human Pilot Study

2021-02-12

Abstract excerpt

<title>Abstract</title> Expansions of a G4C2 repeat in the C9ORF72 gene are the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), two devastating adult-onset neurodegenerative disorders. Proposed disease mechanisms include a gain of toxic functions of the G4C2 repeats, implying that selective reduction in levels of the repeat-containing transcripts would repres...

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Literature Corpus work: e63c4c81-0942-5be4-9dff-fea17ed2998a
DOI: 10.21203/rs.3.rs-211236/v1

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