作者: Chris Paszty , Catherine M Brion , Elizabeth Manci , H Ewa Witkowska , Mary E Stevens
DOI: 10.1126/SCIENCE.278.5339.876
关键词:
摘要: To create mice expressing exclusively human sickle hemoglobin (HbS), transgenic α-, γ-, and β S -globin were generated bred with knockout that had deletions of the murine α- β-globin genes. These cell have major features (irreversibly sickled red cells, anemia, multiorgan pathology) found in humans disease and, as such, represent a useful vivo system to accelerate development improved therapies for this common genetic disease.