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 ; ger
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA DERIVATIVES THEREOF ENZYMOLOGY METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING NUCLEIC ACIDS NUCLEOSIDES NUCLEOTIDES ORGANIC CHEMISTRY PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS SUGARS 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.