Knockout-transgenic mouse model of sickle cell disease

Academic Article


  • When transgenic mice that expressed human sickle hemoglobin were mated with mice having knockout mutations of the mouse α- and β-globin genes, animals were produced that synthesized only human hemoglobin in adult red blood cells. Similar to many human patients with sickle cell disease, the mice developed a severe hemolytic anemia and extensive organ pathology. Numerous sickled erythrocytes were observed in peripheral blood. Although chronically anemic, most animals survived for 2 to 9 months and were fertile. Drug and genetic therapies can now be tested in this mouse model of sickle cell disease.
  • Published In

  • Science  Journal
  • Digital Object Identifier (doi)

    Author List

  • Ryan TM; Ciavatta DJ; Townes TM
  • Start Page

  • 873
  • End Page

  • 876
  • Volume

  • 278
  • Issue

  • 5339