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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Hauptverfasser:	LIU, Yuting, JUNG, Yun Joon, SEHGAL, Alfica, MATTHEWS, Bryan J, CARAVELLA, Justin A, PAI, Rutuja Sudhakar, KELKAR, Rachana S, ROY, Subhadeep, BUMCROT, David A, GAMBOA, Mario Esteban Contreras, SMITH, Cynthia M, AKERBERG, Brynn Nicole-Yoshiko
Format:	Patent
Sprache:	eng ; fre
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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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. L'invention concerne des méthodes de modulation de la transcription du gène OTC à l'aide d'oligonucléotides antisens (ASO) ciblant des ARN régulateurs, tels que des ARN associés au promoteur et des ARN activateurs. Ces méthodes sont utiles pour augmenter l'expression de l'ornithine transcarbamylase (OTC), ce qui permet de traiter des maladies associées à une expression génique aberrante.