Transgenic knockout mice with exclusively human sickle hemoglobinand sickle cell disease

Journal Article · Fri Jun 13 00:00:00 EDT 1997 · Science

OSTI ID:900632

Paszty, C; Brion, C; Manci, E; Witkowska, E; Stevens, M; Narla, M; Rubin, E

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: Lawrence Berkeley National Lab. (LBNL), Berkeley, CA (United States)
• 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: DE-AC02-05CH11231
• OSTI ID: 900632
• Report Number(s): LBNL-40891; SCEHDK; TRN: US200711%%368
• Journal Information: Science, Vol. 278, Issue 5339; Related Information: Journal Publication Date: 1997; ISSN 0193-4511
• Country of Publication: United States
• Language: English

Related Subjects

59 BASIC BIOLOGICAL SCIENCES
DISEASES
GENES
GENETICS
HEMOGLOBIN
IN VIVO
MICE
PATHOLOGY
TRANSGENIC MICE