COMPOSITIONS AND METHODS FOR HYDROXYACID OXIDASE 1 ( HAO1) GENE EDITING FOR TREATING PRIMARY HYPEROXALURIA TYPE 1 (PH1)

Compositions and methods for editing, e.g., introducing double-stranded breaks, within the HAO1 gene are provided. Compositions and methods for treating subjects having primary hyperoxaluria type 1 (PH1), are provided. Se proporcionan composiciones y métodos para editar, por ejemplo, introducir rotu...

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Hauptverfasser:	ODATE Shobu, DYMEK Zachary William, LESCARBEAU Reynald Michael, STRAPPS Walter, MURRAY Bradley Andrew, HESSE Sarah Beth, HUEBNER Anette
Format:	Patent
Sprache:	eng ; spa
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
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Beschreibung
Zusammenfassung:	Compositions and methods for editing, e.g., introducing double-stranded breaks, within the HAO1 gene are provided. Compositions and methods for treating subjects having primary hyperoxaluria type 1 (PH1), are provided. Se proporcionan composiciones y métodos para editar, por ejemplo, introducir roturas bicatenarias, dentro del gen HAO1. Se proporcionan composiciones y métodos para tratar sujetos que tienen hiperoxaluria primaria tipo 1 (PH1).