Blood Diseases

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Blood diseases
  • A molecular switch called BCL11a may hold the key to forcing the red blood cells of patients with sickle cell disease to stop making the mutated adult form of hemoglobin and instead produce the fetal form, which is unaffected by the sickle cell mutation. Read more.
  • Gene therapy holds great promise for correcting Fanconi anemia (FA), a rare, inherited bone marrow failure disease. New insights into why cells from patients with FA resist efforts to reprogram them into induced pluripotent stem cells could help bring the promise closer to reality. Read more.
  • One of the main clinical features of sickle cell disease is the intense pain associated with veno-occlusive crises. A clinical trial based on a new model of how these crises occur could result in a new, targeted way to put a halt to the pain. Read more.
  • A new trial is investigating stem cell transplantation without the use of irradiation of the recipient in FA patients with bone marrow failure. Read more.
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