polyG aggregation

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Antisense oligonucleotides targeting ggc

downin human cells1

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Antisense oligonucleotides targeting ggc decreases polyG aggregation in human cells.
“antisense oligonucleotides targeting GGC effectively reduce polyG aggregation and ameliorate related molecular defects”
Cellsin NIID patient-derived brain organoids
ASO therapy rescues NOTCH2NLC GGC repeat expansion-induced genomic damage, 3D chromatin structural abnormalities, and senescence
Nature communications7 Apr 2026

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Nature Communications7 Apr

ASO therapy rescues NOTCH2NLC GGC repeat expansion-induced genomic damage, 3D chromatin structural abnormalities, and senescence

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