METHODS AND COMPOSITIONS FOR MODULATING PCSK9 EXPRESSION

The present disclosure is directed to compositions and methods for reducing expression of the PCSK9 gene in a cell, e.g., using an expression repressor that comprises a targeting moiety that binds a PCSK9 promoter, anchor sequence, or super-enhancer and an effector domain that represses transcriptio...

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Hauptverfasser:	FARELLI, Jeremiah D, O'DONNELL, Charles W, MORRISON-SMITH, Chevaun Danielle
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 ORGANIC CHEMISTRY PEPTIDES PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS SPIRITS VINEGAR WINE
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creator	FARELLI, Jeremiah D O'DONNELL, Charles W MORRISON-SMITH, Chevaun Danielle
description	The present disclosure is directed to compositions and methods for reducing expression of the PCSK9 gene in a cell, e.g., using an expression repressor that comprises a targeting moiety that binds a PCSK9 promoter, anchor sequence, or super-enhancer and an effector domain that represses transcription or methylates DNA. Systems comprising two or more expression repressors are also disclosed. The compositions can be used, for example, to treat a PCSK9- associated disease or disorder. La présente invention concerne des compositions et des procédés pour réduire l'expression du gène PCSK9 dans une cellule, par exemple, à l'aide d'un répresseur d'expression qui comprend une fraction de ciblage se liant à un promoteur PCSK9, une séquence d'ancrage ou un super-activateur et un domaine effecteur qui réprime la transcription ou méthylate l'ADN. L'invention concerne également des systèmes comprenant au moins deux répresseurs d'expression. Les compositions peuvent être utilisées, par exemple, pour traiter une maladie ou un trouble associés à PCSK9.
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subjects	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING ORGANIC CHEMISTRY PEPTIDES PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS SPIRITS VINEGAR WINE
title	METHODS AND COMPOSITIONS FOR MODULATING PCSK9 EXPRESSION
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