LYSOSOMAL TARGETING OF ENZYMES, AND USES THEREOF
The invention provides compositions and methods for effective lysosomal targeting mediated by PCSK9. In particular, the compositions and methods provided by the invention may be used to treat lysosomal storage diseases such as Pompe Disease and Sanfilippo Syndrome Type B, and they may be used for ta...
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Format | Patent |
Language | English French |
Published |
28.04.2016
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Abstract | The invention provides compositions and methods for effective lysosomal targeting mediated by PCSK9. In particular, the compositions and methods provided by the invention may be used to treat lysosomal storage diseases such as Pompe Disease and Sanfilippo Syndrome Type B, and they may be used for targeting lysosomal enzymes to the various muscles of the human body.
L'invention concerne des compositions et des méthodes de ciblage lysosomal efficace à médiation par PCSK9. En particulier, les compositions et les méthodes selon la présente invention peuvent être utilisées pour traiter des maladies de stockage lysosomal comme la maladie de Pompe et le syndrome de Sanfilippo de type B, et elles peuvent être utilisées pour cibler des enzymes lysosomales sur les divers muscles du corps humain. |
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AbstractList | The invention provides compositions and methods for effective lysosomal targeting mediated by PCSK9. In particular, the compositions and methods provided by the invention may be used to treat lysosomal storage diseases such as Pompe Disease and Sanfilippo Syndrome Type B, and they may be used for targeting lysosomal enzymes to the various muscles of the human body.
L'invention concerne des compositions et des méthodes de ciblage lysosomal efficace à médiation par PCSK9. En particulier, les compositions et les méthodes selon la présente invention peuvent être utilisées pour traiter des maladies de stockage lysosomal comme la maladie de Pompe et le syndrome de Sanfilippo de type B, et elles peuvent être utilisées pour cibler des enzymes lysosomales sur les divers muscles du corps humain. |
Author | ZHANG, BOHONG CONCINO, MICHAEL, F |
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DocumentTitleAlternate | CIBLAGE LYSOSOMAL D'ENZYMES ET UTILISATIONS ASSOCIÉES |
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RelatedCompanies | SHIRE HUMAN GENETIC THERAPIES, INC |
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Snippet | The invention provides compositions and methods for effective lysosomal targeting mediated by PCSK9. In particular, the compositions and methods provided by... |
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SubjectTerms | 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 SPIRITS VINEGAR WINE |
Title | LYSOSOMAL TARGETING OF ENZYMES, AND USES THEREOF |
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