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 α galactosidase A (α-Gal A) protein activity in a subject in need thereof by administering the expression vectors (e.g., a...
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| creator | SOUBERBIELLE, Bernard PASSALACQUA, Cristobal HUSTON, Marshall |
| description | 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 α galactosidase A (α-Gal A) protein activity in a subject in need thereof by administering the expression vectors (e.g., an AAV expression vector) comprising an α galactosidase A (α-Gal A) expression cassette, which comprises an α galactosidase A (α-Gal A) transgene encoding the at least one α-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. |
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| subjects | 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 |
| title | METHODS FOR USE OF VIRAL VECTOR CONSTRUCTS FOR THE TREATMENT OF FABRY DISEASE |
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