New therapeutics for anaemia, haemophilia, thalassemia, sickle cell disease, bone marrow transplantation, hematopoietic stem cell transplantation, thrombocytopenia, pancytopenia.
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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 anaemia. Anaemia currently is treated with a hormone called Epo and its pharmaceutical derivatives. However, many kinds of anaemia 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 anaemia, haemophilia, 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.