Targeted erythropoietin selectively stimulates red blood cell expansion in vivo

Abstract

Erythropoietin is used to treat anemia but has prothrombotic side effects that limit its use. We have demonstrated in vivo the ability to target erythropoietin to red blood cell precursors and away from platelet precursors, thereby potentially avoiding off-target effects. We have systematically determined the protein design features required for in vivo success of the engineered protein. Our results reveal how rational engineering of protein drugs can be used to reduce side effects, with broad implications for designers of therapeutic signaling systems.

Document Details

Document Type
Pub Defense Publication
Publication Date
Apr 25, 2016
Source ID
10.1073/pnas.1525388113

Entities

People

  • Andyna Vernet
  • Devin R. Burrill
  • James J. Collins
  • Jeffrey C Way
  • Pamela Silver

Organizations

  • Broad Institute
  • Harvard Medical School
  • Harvard University
  • Massachusetts Institute of Technology
  • National Heart, Lung, and Blood Institute
  • National Institute of General Medical Sciences
  • Wyss Institute for Biologically Inspired Engineering

Tags

Fields of Study

  • Medicine

Readers

  • Electrical Engineering
  • Immunology
  • Molecular Genetics