Wisconsin Alumni Research Foundation

Pharmaceuticals & Vitamin D
Pharmaceuticals Vitamin D
Increasing Red Blood Cell Production
WARF: P130326US02

Inventors: Emery Bresnick, Yoon A Kang, Skye McIver

The Wisconsin Alumni Research Foundation (WARF) is seeking commercial partners interested in developing a method to enhance erythropoiesis in individuals with anemia, sickle cell disease, hemophilia or other disorders marked by diminished red blood cells.
Overview
Maintaining an adequate supply of oxygen to tissue is necessary for survival. The oxygen-carrying capacity of blood is governed by the concentration of red blood cells (erythrocytes). It is critical that red blood cell production (erythropoiesis) is properly regulated.

A drop in red blood cells due to age, infection, cancer or other condition can cause anemia. Anemia currently is treated with a hormone called Epo and its pharmaceutical derivatives. However, many kinds of anemia are Epo-insensitive. Moreover, treatment with Epo can cause dangerous side effects such as stroke.

Clearly needed is a new method for stimulating red blood cell production.
The Invention
UW–Madison researchers have developed a method for increasing the production of red blood cells by knocking down certain components of the exosome (a protein complex inside cells that degrades RNA). Specifically, the new method uses an siRNA, ribozyme or other inhibitory nucleic acid molecule to decrease the expression of Exosc8, Exosc9, Dis3, Dis3L or Exosc10.

Knocking down these exosome components boosts genes and proteins that promote red blood cell development.
Applications
  • New therapeutics for anemia, hemophilia, thalassemia, sickle cell disease, bone marrow transplantation, hematopoietic stem cell transplantation, thrombocytopenia, pancytopenia or hypoxia
Key Benefits
  • New strategy to encourage red blood cell production
  • Potential alternative for treating Epo-insensitive anemia
  • No known deleterious effects on cell function
Stage of Development
The researchers have shown that siRNA and shRNA are effective at increasing red blood cell maturation.
Additional Information
For More Information About the Inventors
For current licensing status, please contact Andy DeTienne at [javascript protected email address] or 608-960-9857

WARF