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Transgenic knockout mice with exclusively human sickle hemoglobin and sickle cell disease.

To create mice expressing exclusively human sickle hemoglobin (HbS), transgenic mice expressing human alpha-, gamma-, and betaS-globin were generated and bred with knockout mice that had deletions of the murine alpha- and beta-globin genes. These sickle cell mice have the major features (irreversibly sickled red cells, anemia, multiorgan pathology) found in humans with sickle cell disease and, as such, represent a useful in vivo system to accelerate the development of improved therapies for this common genetic disease.

Pubmed ID: 9346488


  • P√°szty C
  • Brion CM
  • Manci E
  • Witkowska HE
  • Stevens ME
  • Mohandas N
  • Rubin EM


Science (New York, N.Y.)

Publication Data

October 31, 1997

Associated Grants

  • Agency: NHLBI NIH HHS, Id: HL20985
  • Agency: NHLBI NIH HHS, Id: HL31579
  • Agency: NHLBI NIH HHS, Id: N01-HB-07086

Mesh Terms

  • Anemia, Sickle Cell
  • Animals
  • Disease Models, Animal
  • Female
  • Globins
  • Hemoglobin, Sickle
  • Humans
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Mice, Knockout
  • Mice, Transgenic