Targeted protein replacement for the treatment of lysosomal storage disorders

摘要

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.

主权项

1. A composition comprising a mammalian acid sphingomyelinase or an active fragment thereof which is attached to a targeting moiety, wherein the targeting moiety binds to an extracellular portion of Intercellular Adhesion Molecule-1 (ICAM-1) or Platelet Endothelial Cell Adhesion Molecule-1 (PECAM-1).