Fanconi anemia (FA) is a life-threatening blood disorder characterized by multiple physical abnormalities, bone marrow failure, and a higher risk of leukemia. It is an inherited disease caused by the mutation in at least 15 different genes. Most children suffering from this disease ultimately require a bone marrow transplant, but the procedure is more challenging than in any other disease. Gene therapy is also difficult in FA patient with fragile bone marrow cells.
In a recent study, researchers have found that inhibition of a regulatory protein (Sh2b3/Lnk) restored the ability of the abnormal bone marrow cells and improved the proliferation and survival of blood cells. This new study may open the door for the treatment of children with Fanconi anemia. Source: Medical News
