Science / Medicine : Genetically Altered Mice Mimic Sickle Cell Disease

Scientists reported last week they have created mice with a disorder resembling sickle cell disease, an advance that may remove a major obstacle to developing treatments for the disorder affecting up to 100,000 Americans. A British research team said it has genetically engineered a strain of mice to contain the defective hemoglobin found in blood cells of humans with sickle cell disease. Hemoglobin is a complex compound responsible for carrying oxygen throughout the body.

Reporting in the journal Nature, a team led by Frank Grosveld of Britain’s National Institute for Medical Research said it was able to create two genetically engineered mice containing relatively large amounts of human sickle cell hemoglobin in their red blood cells.

In test-tube experiments, blood cells taken from the mice were “sickled” under conditions that would cause similar deformities in cells taken from humans with sickle cell disease, researchers said. Other tests showed sickling also appeared to be occurring naturally within one of the mice. However, the researchers pointed out a major problem with their “sickle cell” mice is their failure to develop the anemia and other symptoms characteristic of the human disorder.