Transgenic knockout mice with exclusively human sickle hemoglobin and sickle cell disease
C Paszty, CM Brion, E Manci, HE Witkowska… - Science, 1997 - science.org
Science, 1997•science.org
To create mice expressing exclusively human sickle hemoglobin (HbS), transgenic mice
expressing human α-, γ-, and βS-globin were generated and bred with knockout mice that
had deletions of the murine α-and β-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.
expressing human α-, γ-, and βS-globin were generated and bred with knockout mice that
had deletions of the murine α-and β-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.
To create mice expressing exclusively human sickle hemoglobin (HbS), transgenic mice expressing human α-, γ-, and βS-globin were generated and bred with knockout mice that had deletions of the murine α- and β-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.
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