ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR PAH GENE TRANSFER AND METHODS OF USE THEREOF

Provided herein are adeno-associated virus (AAV) compositions that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell, thereby restoring the PAH gene function. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions. L'inv...

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Bibliographische Detailangaben
Hauptverfasser:	WRIGHT, Jason Boke, RUBIN, Hillard, DOLLIVE, Serena Nicole, AHMED, Seemin Seher, SEYMOUR, Albert Barnes
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
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Beschreibung
Zusammenfassung:	Provided herein are adeno-associated virus (AAV) compositions that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell, thereby restoring the PAH gene function. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions. L'invention concerne des compositions de virus adéno-associés (AAV) susceptibles d'exprimer un polypeptide de phénylalanine hydroxylase (HTAP) dans une cellule, ce qui permet de restaurer la fonction du gène de PAH. L'invention concerne également des procédés d'utilisation des compositions d'AAV et des systèmes de conditionnement permettant de produire les compositions d'AAV.