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

Provided herein are adeno-associated virus (AAV) compositions that can express an arylsulfatase A (ARSA) polypeptide in a cell, thereby restoring the ARSA gene function. Also provided are methods of using the AAV compositions, and packaging systems for making the AAV compositions. L'invention c...

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Bibliographische Detailangaben
Hauptverfasser:	ST. MARTIN, THIA BABOVAL, SEYMOUR, ALBERT BARNES, RUBIN, HILLARD
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 ORGANIC CHEMISTRY PEPTIDES 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 an arylsulfatase A (ARSA) polypeptide in a cell, thereby restoring the ARSA gene function. Also provided are methods of using the AAV compositions, and packaging systems for making the AAV compositions. L'invention concerne des compositions de virus adéno-associés (AAV) qui peuvent exprimer un polypeptide d'arylsulfatase A (ARSA) dans une cellule, ce qui permet de rétablir la fonction du gène ARSA. L'invention concerne également des procédés d'utilisation des compositions d'AAV, et des systèmes d'emballage pour la production des compositions d'AAV.