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:	GALINDO, SILVIA, MURO, SCHUCHMAN, EDWARD, HOWARD, MUZYKANTOV, VLAMIDIR, R
Format:	Patent
Sprache:	eng ; fre
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 MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING NUCLEIC ACIDS NUCLEOSIDES NUCLEOTIDES ORGANIC CHEMISTRY PEPTIDES 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 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 10 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. La présente invention concerne des compositions et des méthodes de transport de protéines lysosomales. Les compositions et les méthodes de l'invention permettent d'acheminer de façon ciblée des protéines lysosomales exogènes vers des protéines de surface permettant leur internalisation par des voies autres que celles de la clathrine. L'invention concerne également l'utilisation desdites compositions et méthodes destinées au traitement par remplacement enzymatique de maladies lysosomales. Elle concerne en outre des acides nucléiques, des cellules recombinantes, et des trousses utiles servant à produire et mettre en oeuvre les compositions de l'invention.