METHODS OF TREATING PHENYLKETONURIA

Provided herein are methods of treating a subject having a disease or disorder associated with a PAH gene mutation. The methods generally comprise administering to the subject a therapeutically effective does of a recombinant adeno-associated virus (rAAV) that can express a phenylalanine hydroxylase...

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Hauptverfasser:	LAMPPU, Diana, WRIGHT, Teresa, SEYMOUR, Albert, FRANCONE, Omar, KINCH, Deborah
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
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Beschreibung
Zusammenfassung:	Provided herein are methods of treating a subject having a disease or disorder associated with a PAH gene mutation. The methods generally comprise administering to the subject a therapeutically effective does of a recombinant adeno-associated virus (rAAV) that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell and thereby restore PAH gene function in the subject. L'invention concerne des procédés de traitement d'un sujet souffrant d'une maladie ou d'un trouble associé à une mutation du gène PAH Les procédés comprennent généralement l'administration au sujet d'une dose thérapeutiquement efficace d'un virus adéno-associé recombinant (rAAV) qui peut exprimer un polypeptide de phénylalanine hydroxylase (PAH) dans une cellule et ainsi restaurer la fonction du gène PAH chez le sujet.