Regulation of gene transcription using antisense oligonucleotides targeted to modulate RNA

Described herein are methods of regulating OTC gene transcription using antisense oligonucleotides (ASOs) that target regulatory RNAs (e.g., promoter-related RNAs and enhancer RNAs). These methods are useful for increasing the expression of ornithine transcarbamoylase (OTC), thereby treating disease...

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Hauptverfasser:	SMITH CHARLENE MARIE, KELKAR RAJESH S, CHUNG YEON-JUN, GAMBOA MATTHEW E. C, LIU YUTING, BUMCROT DAVID A, AKERBERG BEN NILS-YVONNE, SEHGAL ALFICA, ROY SASHWATI, MATTHEWS BRYAN J, PAI RAJESH S, CARAVELLA JUSTIN A
Format:	Patent
Sprache:	chi ; eng
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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Beschreibung
Zusammenfassung:	Described herein are methods of regulating OTC gene transcription using antisense oligonucleotides (ASOs) that target regulatory RNAs (e.g., promoter-related RNAs and enhancer RNAs). These methods are useful for increasing the expression of ornithine transcarbamoylase (OTC), thereby treating diseases associated with abnormal gene expression. 本文描述了使用靶向调节RNA(例如启动子相关RNA和增强子RNA)的反义寡核苷酸(ASO)调控OTC基因转录的方法。这些方法可用于增加鸟氨酸转氨甲酰酶(OTC)的表达，从而治疗与异常基因表达相关的疾病。