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Antisense Oligonucleotides Targeting an <i>LDLR</i> Regulatory RNA Increase <i>LDLR</i> Expression and Reduce LDL-cholesterol in Vivo

2026-06-04

Abstract excerpt

Regulatory RNAs (regRNAs) are non-coding RNAs expressed from promoters and enhancers that modulate gene transcription. Targeting regRNAs with antisense oligonucleotides (ASOs) can increase transcription and presents a potential therapeutic strategy to restore expression in haploinsufficient diseases. This approach requires high-quality regRNA maps, identification of ASOs targeting actionable sequences, and transla...

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Literature Corpus work
a6d3bb5d-983c-5dc2-9cc2-ac8a96e42739
DOI
10.64898/2026.06.02.729508
Open publication

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Antisense Oligonucleotides Targeting an <i>LDLR</i> Regulatory RNA Increase <i>LDLR</i> Expression and Reduce LDL-cholesterol in VivoDOI 10.64898/2026.06.02.729508
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