COMPOSITIONS, SYSTEMS, AND METHODS FOR TREATING FAMILIAL HYPERCHOLESTEROLEMIA BY TARGETING PCSK9

Described herein are compositions, systems, and methods for modulating gene expression. Also described herein are systems and methods for treating a disease or a condition by modulating gene expression. In some embodiments, the compositions, systems, and methods provided herein are used to treat fam...

Ausführliche Beschreibung

Gespeichert in:

Bibliographische Detailangaben
Hauptverfasser:	LIU, Yanxia, KO, Tengyu, JADHAV, Kavita, BOREGOWDA, Siddaraju Valagerehalli, COLLIN DE L'HORTET, Alexandra Sylvie, PHAM, Annie, NORTON, Andrew Joseph, SALZMAN, Amber Ruth, YANG, Guang
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 SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS SPIRITS VINEGAR WINE
Online-Zugang:	Volltext bestellen
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!

Beschreibung
Zusammenfassung:	Described herein are compositions, systems, and methods for modulating gene expression. Also described herein are systems and methods for treating a disease or a condition by modulating gene expression. In some embodiments, the compositions, systems, and methods provided herein are used to treat familial hypercholesterolemia. In some embodiments, the compositions, systems, and methods provided herein involve suppression of endogenous PCSK9. L'invention concerne des compositions, des systèmes et des procédés de modulation de l'expression génique. L'invention concerne également des systèmes et des méthodes de traitement d'une maladie ou d'une affection par modulation de l'expression génique. Dans certains modes de réalisation, les compositions, les systèmes et les procédés de l'invention sont utilisés pour traiter l'hypercholestérolémie familiale. Dans certains modes de réalisation, les compositions, les systèmes et les procédés de l'invention impliquent la suppression de PCSK9 endogène.