Professor Yuet Wai Kan and his team from University of California, San Francisco using gene therapy has replaced the genes responsible for causing sickle cell anemia in bioengineered mice with healthy stem cells from embryos leading to replacement of defective genes, after therapy it was found that the replaced healthy genes produces non-defective healthy hemoglobin. The researchers have published their work in the Journal Proceedings of the National Academy of Sciences.
Sickle cell anemia is a hereditary disorder in persons who have two abnormal genes that makes their red blood cells (RBC) appear crescent moon shaped. The hemoglobin which carries oxygen in blood is affected by this hereditary disease leading to abnormal hemoglobin form, which mostly clumps together and makes the blood sticky and gets clogs in blood stream and these blood cells becomes very fragile leading to break down within 10 to 20 days leading to anemia.
Researchers have used embryonic stem cells for treating mutations in human sickle cell anemia by inserting healthy hemoglobin gene, though the work has been successful in bioengineered mice containing human genes it has to be done in human clinical trial. Gene therapy and stem cell therapy are new treatment options which show great avenues in treating genetic disorders such as sickle cell anemia, Thalassemia, hemophilia and other genetic diseases.