OLIGONUCLEOTIDE PROGRANULIN AGONISTS

The present invention relates to oligonucleotides that upregulate or restore the expression of progranulin in cells by targeting the promoter region of the progranulin gene. The invention further relates to pharmaceutical compositions and methods for the treatment of diseases associated with progran...

Ausführliche Beschreibung

Gespeichert in:

Bibliographische Detailangaben
Hauptverfasser:	BRAUN, Johannes, RASMUSSEN, Søren V, JOENSON, Lars, WORM, Jesper, VANG, Dorthe
Format:	Patent
Sprache:	eng ; fre
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
Online-Zugang:	Volltext bestellen
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!

Beschreibung
Zusammenfassung:	The present invention relates to oligonucleotides that upregulate or restore the expression of progranulin in cells by targeting the promoter region of the progranulin gene. The invention further relates to pharmaceutical compositions and methods for the treatment of diseases associated with progranulin, specifically progranulin haploinsufficiency and neurological disorders. La présente invention concerne des oligonucléotides qui régulent à la hausse ou restaure l'expression de la progranuline dans des cellules par ciblage de la région de promoteur du gène de la progranuline. L'invention concerne en outre des compositions pharmaceutiques et des méthodes pour le traitement de maladies associées à la progranuline, spécifiquement l'haploinsuffisance de progranuline et les troubles neurologiques.