COMPOSITIONS AND METHODS FOR INTERNALIZING ENZYMES

Compositions and methods for treating lysosomal storage diseases are disclosed. Biotherapeutic complexes containing an internalization effector binding domain and a lysosomal replacement enzyme activity are disclosed. The biotherapeutic complexes are capable of entering cells, segregating to the lys...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: Cygnar, Katherine, Baik, Andrew
Format: Patent
Sprache:eng
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Beschreibung
Zusammenfassung:Compositions and methods for treating lysosomal storage diseases are disclosed. Biotherapeutic complexes containing an internalization effector binding domain and a lysosomal replacement enzyme activity are disclosed. The biotherapeutic complexes are capable of entering cells, segregating to the lysosome, and delivering the replacement enzyme activity to the lysosome.