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

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, ...

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.

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 ...

Sickle cell disease is caused by a genetic mutation in the hemoglobin within red blood cells. This abnormal hemoglobin sticks together, changing the shape of a person's red blood cells.

In 2021 sickle cell disease was one of the top three causes of ... The main genetic mutation known to keep HbF levels higher after birth is within BCL11A, but that mutation accounts for only a ...

When Vijay Sankaran was an MD-PhD student at Harvard Medical School in the mid-2000s, one of his first clinical encounters was with a 24-year-old patient whose sickle cell disease left them with ...

With sickle cell disease, the mutations in a person’s two copies of the HBB gene cause red blood cells to become rigid and crescent-shaped, resembling a sickle, rather than the usual round ...

Families of those who have sickle cell disease in the Philadelphia area are pushing for more research and a cure.

In sickle cell disease, red blood cells become "sickle" or crescent-shaped due to a genetic mutation affecting hemoglobin -- a protein that carries oxygen from the lungs to other tissues.

UCSF Benioff Children’s Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 ...