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News-in-brief / 05.12.15

Study explores role of BCL11A in neurodevelopment

By Broad Communications

Fetal hemoglobin, which is normally replaced by adult hemoglobin a few months after birth, can ameliorate symptoms of beta-thalassemia and sickle cell anemia. Boosting fetal hemoglobin by inhibiting the transcription factor BCL11A holds therapeutic promise, but BCL11A's role in the body isn’t fully understood. To study its in vivo effects, a team led by Broad associate member Vijay Sankaran, Mark Daly, co-director of the Broad’s Medical and Population Genetics Program, and Zdenek Sedlacek of University Hospital Motol (Czech Republic) identified and characterized three patients with an autism spectrum disorder and developmental delay who harbored deletions of the BCL11A gene. The study, appearing in the Journal of Clinical Investigation, provides evidence of BCL11A’s role in neurodevelopment and suggests caution when developing BCL11A-targeting therapies.