MODULATION OF GENE TRANSCRIPTION USING ANTISENSE OLIGONUCLEOTIDES TARGETING REGULATORY RNAS

Described herein are methods of modulating gene transcription using antisense oligonucleotides (ASOs) targeting regulatory RNAs, such as promoter-associated RNAs and enhancer RNAs. These methods are useful for modulating the levels of gene products, for example, increasing expression of Carbamoyl-Ph...

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Hauptverfasser: KELKAR, RACHANA S, BUMCROT, DAVID A, SEHGAL, ALFICA, MATTHEWS, BRYAN J, GAMBOA, MARIO ESTEBAN CONTRERAS, LIU, YUTING, ROY, SUBHADEEP, MOUKHEIBER, HENRY MUNIR, GUO, YUCHUN, CARAVELLA, JUSTIN A, PAI, RUTUJA SUDHAKAR, JUNG, YUN JOON
Format: Patent
Sprache:eng ; fre
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Zusammenfassung:Described herein are methods of modulating gene transcription using antisense oligonucleotides (ASOs) targeting regulatory RNAs, such as promoter-associated RNAs and enhancer RNAs. These methods are useful for modulating the levels of gene products, for example, increasing expression of Carbamoyl-Phosphatase Synthetase 1 (CPS1), thereby treating diseases associated with aberrant gene expression. L'invention concerne des méthodes de modulation de la transcription génique à l'aide d'oligonucléotides antisens (ASO) ciblant des ARN régulateurs, tels que des ARN associés au promoteur et des ARN amplificateurs. Ces méthodes sont utiles pour moduler les niveaux de produits géniques, par exemple, augmenter l'expression de carbamoyl-phosphatase synthétase 1 (CPS1), ce qui permet de traiter des maladies associées à une expression génique aberrante.