LYSOSOMAL POLYPEPTIDES, METHODS OF MAKING AND USING

摘要

The invention generally relates to compositions and methods for producing lysosomal proteins that have altered glycan structure, such that the protein can be delivered efficiently into the lysosomes of target cells (such as macrophages). The lysosomal proteins are produced by modifying the glycosylation pathways in a host cell using an RNA effector molecule, such as an siRNA. Glycan-modified lysosomal proteins produced using the methods described herein have improved properties, including e.g., increased specific uptake by target cells (such as macrophages).