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Transgenic knockout mice with exclusively human sickle hemoglobinand sickle cell disease

Journal Article · · Science
OSTI ID:900632
To create mice expressing exclusively human sicklehemoglobin (HbS), transgenic mice expressing human alpha-, gamma-, andbeta[S]-globin were generated and bred with knockout mice that haddeletions of the murine alpha- and beta-globin genes. These sickle cellmice have the major features (irreversibly sickled red cells, anemia,multiorgan pathology) found in humans with sickle cell disease and, assuch, represent a useful in vivo system to accelerate the development ofimproved therapies for this common genetic disease.
Research Organization:
Ernest Orlando Lawrence Berkeley NationalLaboratory, Berkeley, CA (US)
Sponsoring Organization:
USOER; National Heart Lung and Blood Institute (NHLBI)grants HL31579 and HL20985 to the Northern California ComprehensiveSickle Cell Cente r, A Red Cell Program Project Grant (DK32094) and fromthe National Inst. of Diabetes, Digestive and Kidney Diseases, a NHLBIcontract to the Sickle Cell Disease Centralized Pathology Unit and an NIHShared Instrume
DOE Contract Number:
AC02-05CH11231
OSTI ID:
900632
Report Number(s):
LBNL--40891
Journal Information:
Science, Journal Name: Science Journal Issue: 5339 Vol. 278; ISSN 0193-4511; ISSN SCEHDK
Country of Publication:
United States
Language:
English

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