Targeted protein replacement for the treatment of lysosomal storage disorders

The present invention relates to compositions and methods for delivering lysosomal proteins. The compositions and methods described herein permit the targeted delivery of exogenous lysosomal proteins to cell surface proteins that allow their internalization via non-clathrin pathways. The present inv...

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Bibliographische Detailangaben
Hauptverfasser:	SCHUCHMAN EDWARD HOWARD, MURO GALINDO SILVIA, MUZYKANTOV VLADIMIR R
Format:	Patent
Sprache:	eng
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA DERIVATIVES THEREOF ENZYMES ENZYMOLOGY GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS HUMAN NECESSITIES HYGIENE LAYERED PRODUCTS LAYERED PRODUCTS, i.e. PRODUCTS BUILT-UP OF STRATA OF FLAT ORNON-FLAT, e.g. CELLULAR OR HONEYCOMB, FORM MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING NUCLEIC ACIDS NUCLEOSIDES NUCLEOTIDES ORGANIC CHEMISTRY PEPTIDES PERFORMING OPERATIONS PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS SUGARS TECHNICAL SUBJECTS COVERED BY FORMER US CLASSIFICATION TECHNICAL SUBJECTS COVERED BY FORMER USPC TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS TRANSPORTING VINEGAR WINE
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Beschreibung
Zusammenfassung:	The present invention relates to compositions and methods for delivering lysosomal proteins. The compositions and methods described herein permit the targeted delivery of exogenous lysosomal proteins to cell surface proteins that allow their internalization via non-clathrin pathways. The present invention further relates to the use of the compositions and methods for enzyme replacement therapy of lysosomal storage diseases. Nucleic acids, recombinant cells and kits useful for making and using the compositions of the invention are also provided.