TARGETED THERAPEUTIC PROTEINS

摘要

Targeted therapeutics that localize to a specific subcellular compartment such as the lysosome are provided. The targeted therapeutics include a therapeutic agent and a targeting moiety that binds a receptor on an exterior surface of the cell, permitting proper subcellular localization of the targeted therapeutic upon internalization of the receptor. Nucleic acids, cells, and methods relating to the practice of the invention are also provided.

主权项

1. A targeted therapeutic comprising:
a therapeutic agent that is therapeutically active in a mammalian lysosome; and means for binding an extracellular domain of human cation-independent mannose-6-phosphate receptor in a mannose-6-phosphate-independent manner.