Article

Dual-targeting CRISPR-CasRx reduces <i>C9orf72</i> ALS/FTD sense and antisense repeat RNAs in vitro and in vivo

2024-01-28

Abstract excerpt

The most common genetic cause of both frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) is a G 4 C 2 repeat expansion in intron 1 of the C9orf72 gene. This repeat expansion undergoes bidirectional transcription to produce sense and antisense repeat RNA species. Both sense and antisense-derived repeat RNAs undergo repeat-associated non-AUG translation in all reading frames to generate five di...

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Literature Corpus work: 8f8fa183-cfed-5003-9e70-7b6bc9760b95
DOI: 10.1101/2024.01.26.577366

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