Thalassemia Models
α - Thalassemia is an inherited blood disorder characterized by reduced α-globin synthesis, resulting in an imbalance between α and β globin chains and leading to increased levels of HbH and Hb Barts. Its clinical severity is determined by the number of functional α-globin genes. Β-Thalassemia, on the other hand, is caused by reduced or absent β-globin chain synthesis, causing an excess of α chains which form inclusion bodies in red blood cells, leading to oxidative damage and ineffective erythropoiesis.
β-Thalassemia can take several different forms: carriers (β/β+ or β/β0) usually have mild to moderate microcytic anemia and are often asymptomatic; intermedia (β+/β+ or β+/β0) may not need, or only occasionally need, blood transfusions; and major (β0/β0 or β+/β0) presents as severe transfusion-dependent anemia (TDT) with symptoms like severe anemia, hepatosplenomegaly, and growth retardation.
● Genetically Engineered Thalassemia Mouse Models
|
Strain No.
|
Strain Name | Strain Type | Description |
|---|---|---|---|
| T037237 | Hbb-bs-&Hbb-bt-KO | Knockout | Hbb-bs&Hbb-bt-dKO heterozygous mice display reduced hemoglobin, lower MCV and MCH values and significantly increased reticulocytes, which indicates β-thalassemia disease phenotype. Red blood cells in Hbb-bs-KO; Hbb-bt-KO heterozygous mice are abnormal and cytolyzed, and iron deposition is increased in the liver, spleen and kidney of the mice |
| T003802 | NCG-X | Point mutation | NCG-X mice naturally display impaired erythropoiesis, with reduced red blood cell counts and hemoglobin compared to wildtype NCG and B6 mice. Upon inhibition of macrophages, NCG-X is suitable for mature erythrocyte reconstitution and evaluation of HSC-based therapies against thalassemia |
| T059991 | Hba-a1&Hba-a2-KO | Knockout | Hba-a1&Hba-a2-dKO heterozygous mice display reduced hemoglobin, lower MCV and MCH values and significantly increased reticulocytes, which indicates α-thalassemia disease phenotype. Red blood cells in Hbb-bs-KO; Hbb-bt-KO heterozygous mice are abnormal and cytolyzed, and iron deposition is increased in the spleen of the mice |
| T053637 | Hba-x&Hba-a1&Hba-a2-KO(α-thalassemia) | Knockout | Hba-x&Hba-a1&Hba-a2-tKO heterozygous mice display reduced hemoglobin, lower MCV and MCH values and significantly increased reticulocytes, which indicates α-thalassemia disease phenotype |