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Antisense oligonucleotide targeting pathogenic sense repeat RNA in <i>C9ORF72</i> suppresses production of antisense-dependent dipeptide repeat proteins implicated in ALS/FTD

2024-10-05

Abstract excerpt

A six nucleotide repeat expansion in intron-1 of the C9ORF72 gene is the most common genetic mutation affecting individuals with Amyotrophic Lateral Sclerosis and Frontotemporal Dementia. Bi-directional transcription of the repeat expansion generates sense and antisense repeat RNAs that can then be translated in all reading frames to produce six distinct dipeptide repeat (DPR) proteins with unique termini. The pr...

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Literature Corpus work
dd1a65b7-a611-5b73-bcc4-e3866902abce
DOI
10.1101/2024.10.04.616663
Open publication

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