METHODS FOR USE OF VIRAL VECTOR CONSTRUCTS FOR THE TREATMENT OF FABRY DISEASE

The present disclosure relates to the methods for treating or ameliorating one or more symptoms of Fabry disease, reducing the amount of glycosphingolipids, and/or increasing an ? galactosidase A (?-Gal A) protein activity in a subject in need thereof by administering the expression vectors (e.g., a...

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Bibliographische Detailangaben
Hauptverfasser:	HUSTON, MARSHALL, SOUBERBIELLE, BERNARD, PASSALACQUA, CRISTOBAL
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:	The present disclosure relates to the methods for treating or ameliorating one or more symptoms of Fabry disease, reducing the amount of glycosphingolipids, and/or increasing an ? galactosidase A (?-Gal A) protein activity in a subject in need thereof by administering the expression vectors (e.g., an AAV expression vector) comprising an ? galactosidase A (?-Gal A) expression cassette, which comprises an ? galactosidase A (?-Gal A) transgene encoding the at least one ?-Gal A protein at a dose of about 5×1012 vector genomes per kilogram of body weight (vg/kg) to about 5×1013 vg/kg. La présente divulgation concerne les procédés pour traiter ou atténuer un ou plusieurs symptômes de la maladie de Fabry, réduire la quantité de glycosphingolipides, et/ou augmenter une activité de protéine ? galactosidase A (?-Gal A) chez un sujet en ayant besoin, en administrant les vecteurs d'expression (par ex, un vecteur d'expression AAV) comprenant une cassette d'expression ? galactosidase A (?-Gal A), qui comprend un transgène ? galactosidase A (?-Gal A) codant pour au moins une protéine ?-Gal A à une dose allant d'environ 5×1012 vecteurs génomiques par kilogramme de poids corporel (vg/kg) à environ 5×1013 vg/kg.