Article
BCL11A deletions result in fetal hemoglobin persistence and neurodevelopmental alterations.
The Journal of clinical investigation - 1 Jun 2015
Basak Anindita, Hancarova Miroslava, Ulirsch Jacob C, Balci Tugce B, Trkova Marie, Pelisek Michal, Vlckova Marketa, Muzikova Katerina, Cermak Jaroslav, Trka Jan, Dyment David A, Orkin Stuart H, Daly Mark J, Sedlacek Zdenek, Sankaran Vijay G
Abstract excerpt
A transition from fetal hemoglobin (HbF) to adult hemoglobin (HbA) normally occurs within a few months after birth. Increased production of HbF after this period of infancy ameliorates clinical symptoms of the major disorders of adult β-hemoglobin: β-thalassemia and sickle cell disease. The transcription factor BCL11A silences HbF and has been an attractive therapeutic target for increasing HbF levels; however,...
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