Orkin's team has now reported the crystal structure of the BCL11A tetramer and suggested several ways to attack it with small molecules. "The tetramer is like a blob with nooks and crannies ...

The switch from fetal γ-globin to adult β-globin is controlled by BCL11A. Genetic manipulation of BCL11A improves disease outcomes by turning γ-globin expression back on to compensate for ...

The researchers suspect that BCL11A plays a protective role for ... Labeling with fluorescent molecules. ... 2021 — A new gene therapy targeting the small brain region where dopamine neurons ...

The third possibility, which I and others including Vertex are working on, is small molecules. BCL11A is validated. We know it's a good target.

Small molecules hint at a new treatment option ... Researchers from Mass General Brigham also reported at this year’s ASH meeting that a dual degrader of ZBTB7A and BCL11a appears to boost fetal ...

THE secret to curing sickle cell anaemia could be in our genes. People with the disease have abnormally shaped red blood cells (RBCs) and experience pain and breathlessness as a result.

New small molecule could treat sickle cell disease in adults that don't respond to hydroxyurea, alone. ScienceDaily . Retrieved June 2, 2025 from www.sciencedaily.com / releases / 2024 / 07 ...

Specially designed lengths of RNA, injected into the bloodstream, could bind with the BCL11A gene and silence it. This approach, however, would be expensive and impractical on a large scale.

The researchers suspect that BCL11A plays a protective role for ... They then labeled BCL11A-producing neurons in mice with fluorescent molecules, ... but only a small part of this brain area.