MODULATION OF ABCB11 GENE TRANSCRIPTION USING ANTISENSE OLIGONUCLEOTIDES TARGETING REGULATORY RNAS

Described herein are methods of modulating ABCB11 gene transcription using antisense oligonucleotides (ASOs) targeting regulatory RNAs, such as promoter-associated RNAs and enhancer RNAs. These methods are useful for increasing the expression of ABCB11 mRNA and protein to treat subjects having or at...

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Bibliographische Detailangaben
Hauptverfasser:	GNANAPRADEEPAN, Keerthana, LIU, Yuting, SEHGAL, Alfica, PAI, Rutuja Sudhakar, RODRIGUEZ, Mateo John, KELKAR, Rachana S, SCHWARTZ, Brian E, BUMCROT, David A
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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Beschreibung
Zusammenfassung:	Described herein are methods of modulating ABCB11 gene transcription using antisense oligonucleotides (ASOs) targeting regulatory RNAs, such as promoter-associated RNAs and enhancer RNAs. These methods are useful for increasing the expression of ABCB11 mRNA and protein to treat subjects having or at risk of developing cholestasis or a cholestatic liver disease, such as primary biliary cholangitis and progressive familial intrahepatic cholestasis. La présente invention concerne des procédés de modulation de la transcription du gène ABCB11 à l'aide d'oligonucléotides antisens (ASO) ciblant les ARN régulateurs, tels que les ARN associés au promoteur et les ARN activateurs. Ces procédés sont utiles pour augmenter l'expression de l'ARNm et de la protéine ABCB11 afin de traiter des sujets présentant ou risquant de présenter une cholestase ou une maladie cholestatique hépatique, telles que la cholangite biliaire primitive et la cholestase intrahépatique familiale progressive.