Hematology of a murine. beta. -thalassemia: a longitudinal study

Journal Article · Wed Jan 01 00:00:00 EST 1986 · Ann. N.Y. Acad. Sci.; (United States)

OSTI ID:5677344

Popp, R A; Popp, D M; Johnson, F M; Skow, L C; Lewis, S E

Mice homozygous for a spontaneous mutation, in which the ..beta..-major globin gene is deleted, have clinical symptoms of ..beta..-thalassemia. These mice have a hypocellular, hypochromic, microcytic anemia that becomes more severe with increasing age. The defective red cell morphology, decreased osmotic fragility of erythrocytes and shortened red cell life span found in ..beta..-thalassemic mice are similar to those observed in human ..beta..-thalassemia. Synthesis of ..beta..-globin is depressed but not as much as might be expected because the expression of the..beta..-minor globin gene is enhanced to encode two to three times more globin than in normal mice. Splenomegaly, an enlarged pool of stem cells for erythropoiesis, and iron overloading occur in older mice. The fact that these mice remain moderately healthy makes them a very suitable animal model in which to develop and test alternative techniques of gene therapy that could be successfully applied to the treatment of human thalassemia. Homozygous ..beta..-thalassemic mice have large deposits of iron in their tissues, which might make these mice also useful for in vivo tests of the effectiveness and possible long-term side effects for newly developed iron chelators.

• Research Organization: Oak Ridge National Lab., TN
• DOE Contract Number: AC05-84OR21400
• OSTI ID: 5677344
• Journal Information: Ann. N.Y. Acad. Sci.; (United States), Vol. 445
• Country of Publication: United States
• Language: English

Related Subjects

59 BASIC BIOLOGICAL SCIENCES
ERYTHROCYTES
LIFE SPAN
THALASSEMIA
HEMATOLOGY
AGE DEPENDENCE
BIOLOGICAL MODELS
EXPERIMENTAL DATA
GENES
GLOBIN
MICE
MUTATIONS
ANEMIAS
ANIMALS
BIOLOGICAL MATERIALS
BLOOD
BLOOD CELLS
BODY FLUIDS
DATA
DISEASES
HEMIC DISEASES
INFORMATION
MAMMALS
MATERIALS
MEDICINE
NUMERICAL DATA
ORGANIC COMPOUNDS
PROTEINS
RODENTS
SYMPTOMS
VERTEBRATES
550900* - Pathology