Sickle cell disease is caused by a mutation in hemoglobin, the oxygen-carrying protein in red blood cells, which causes the usually pliant cells to stiffen and assume a curved or "sickled" shape.

Sickle cell disease is caused by a mutation in hemoglobin, the oxygen-carrying protein in red blood cells, ... which is affected by the sickle-cell mutation. So when you knock BCL11A down, ...

Every year about 300,000 babies around the world are born with sickle cell disease, the result of a mutation in the gene for hemoglobin, the oxygen-carrying molecule in red blood cells.

Flipping a single molecular switch can reverse illness in a model of sickle cell disease, according to a study by researchers at Children's Hospital Boston and Dana-Farber Cancer Institute. When ...

Patients with sickle cell disease inherit a gene mutation that produces abnormal hemoglobin, ... CASEVY works by targeting — and "turning off" — the BCL11A gene.

Flipping a single molecular switch can reverse illness in a model of sickle cell disease, according to a new study. When turned off, the switch, a protein called BCL11A, allows the body to ...

The promise of a revolutionary gene-editing technology is beginning to be realized in experiments aimed at curing sickle cell disease. Scientists reported Wednesday that they used the CRISPR-Cas9 ...

In sickle cell disease, a gene mutation causes the hemoglobin molecules in red blood cells to form a rigid chain. ... "The BCL11A protein is the best and most direct target," he says, ...

A direct consequence of natural selection among Homo sapiens, sickle cell disease (SCD) has afflicted a large segment of the population for several millennia.However, with the rapid rise of new ...

In patients with sickle cell disease, there's a single, small mutation in the gene that encodes hemoglobin. The mutation is a switch of a single nucleotide, or base, (often represented by letters ...

To safely target BCL11A for sickle cell disease, the team members identified a region within the gene that’s crucial for BCL11A expression specifically in red blood cells.