Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas

Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas

Described herein are methods of modulating OTC gene transcription using antisense oligonucleotides (ASOs) targeting regulatory RNAs, such as promoter-associated RNAs and enhancer RNAs. These methods are useful for increasing expression of Ornithine transcarbamylase (OTC), thereby treating diseases a...

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Bibliographische Detailangaben
Hauptverfasser: KELKAR Rachana S, BUMCROT David A, SEHGAL Alfica, AKERBERG Brynn Nicole-Yoshiko, GAMBOA Mario Esteban Contreras, ROY Subhadeep, MATTHEWS Bryan J, CARAVELLA Justin A, JUNG Yun Joon, LIU Yuting, SMITH Cynthia M, PAI Rutuja Sudhakar
Format: Patent
Sprache:eng ; heb
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BIOCHEMISTRY
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Zusammenfassung:Described herein are methods of modulating OTC gene transcription using antisense oligonucleotides (ASOs) targeting regulatory RNAs, such as promoter-associated RNAs and enhancer RNAs. These methods are useful for increasing expression of Ornithine transcarbamylase (OTC), thereby treating diseases associated with aberrant gene expression.