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Effective mixed backbone antisense oligonucleotides inhibit the expression of mutant C9orf72

 Last Update: 2022-01-10
 Source: Internet
 Author: User

Summary

The expansion of the G4C2 duplication in the C9ORF72 gene is the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), two devastating adult neurodegenerative diseases
.

Using C9-ALS/FTD patient-derived cells and C9ORF72 BAC transgenic mice, we generated and optimized antisense oligonucleotides (ASOs), which can selectively attenuate the expression of G4C2 repeat transcripts and effectively Inhibit the level of poly (GP) dipeptide in the tissue

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