Sentences with phrase «severe thalassemia»
Also, prenatal diagnosis is available for pregnancies at risk for severe thalassemia.
https://www.scientificamerican.com/ Not exact matches
bluebird bio (BLUE)- The company's BCMA CAR - T drug candidate bb2121 remains attractive despite overblown fears on durability (peak sales of $ 2 billion or more), LentiGlobin has a good shot at success in TDT (Transfusion - Dependent ß - Thalassemia) and SCD (Severe Sickle Cell Disease), and they have a strong cash position.
Last month she filed a request with the U.S. Food and Drug Administration to offer hematopoietic stem cells to fetuses with α thalassemia, a severe form of the disease that is fatal before or soon after birth.
Notably, research groups might be able to apply the approach described in this study to develop treatments for other blood diseases such as β - thalassemia, severe combined immunodeficiency (SCID), chronic granulomatous disease, rare disorders like Wiskott - Aldrich syndrome and Fanconi anemia, and even HIV infection.
A person with b thalassemia major, in which both genes are affected, develops severe anemia starting at a few months of age.
People who have an absence or severe abnormality of only one of their b globin genes have b thalassemia minor.
The most common type in the United States is b thalassemia, also known as Mediterranean anemia in its mild form or Cooley's anemia in its severe form.
Other blood disorders that have shown significant benefit from targeted gene therapy in small trials include hemophilia (specifically, factor IX deficiency), severe beta - thalassemia (deficiency for the adult beta - globin gene) and leukemia, where the patient's immune cells were treated to enable them to recognize cancer cells and destroy them.
The BBC reports that gene therapy has been used successfully to treat a patient with severe β - thalassemia.