Regulation of gene transcription using antisense oligonucleotides targeted to modulate RNA

Described herein are methods of regulating gene transcription using antisense oligonucleotides (ASOs) that target regulatory RNAs (e.g., promoter-related RNAs and enhancer RNAs). These methods are useful for modulating the levels of gene products, such as increasing the expression of carbamyl phosph...

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Hauptverfasser:	KELKAR RAJESH S, CHUNG YEON-JUN, MUKKABEL, HANNAH, M, GAMBOA MATTHEW E. C, LIU YUTING, GUO YINZHONG, BUMCROT DAVID A, 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 ENZYMOLOGY METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	Described herein are methods of regulating gene transcription using antisense oligonucleotides (ASOs) that target regulatory RNAs (e.g., promoter-related RNAs and enhancer RNAs). These methods are useful for modulating the levels of gene products, such as increasing the expression of carbamyl phosphate synthase 1 (CPS1), 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)调控基因转录的方法。这些方法可用于调控基因产物的水平，例如增加氨甲酰磷酸合成酶1(CPS1)的表达，从而治疗与异常基因表达相关的疾病。