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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Bibliographic Details
Main Authors CONCINO, MICHAEL, F, ZHANG, BOHONG
Format Patent
LanguageEnglish
French
Published 28.04.2016
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
SPIRITS
VINEGAR
WINE
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Summary: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.
Bibliography:Application Number: WO2015US57214