Sickle cell disease (SCD) is a group of inherited blood disorders resulting from a mutation in the hemoglobin protein in red blood cells. This mutation changes the shape of the blood cells from round, ...

In sickle cell disease, a gene mutation causes the hemoglobin molecules ... work in all patients and are not very potent. "The BCL11A protein is the best and most direct target," he says, "but ...

Sickle-cell anaemia is the name of a specific form of sickle-cell disease in which there is homozygosity for the mutation that causes HbS. Sickle-cell anaemia is also referred to as "HbSS", "SS ...

The Gates Foundation will invest up to $50 million to support Tessera's SCD gene editing treatment, the company said.

Beam is pursuing a long-term, staged development strategy for sickle cell disease that includes three “waves” of ...

Orna Therapeutics and Vertex will use lipid nanoparticle system to develop gene-editing therapies for people with SCD and TDT ...

Up to 30% of patients with AML have a specific mutation ... a protein called MALT1, a key component of a signaling pathway that chronic lymphocytic leukemia (CLL) and other B-cell cancers depend ...

For the first time, scientists have created a database that details how hundreds of thousands of genetic mutations disrupt the function of proteins, leading to disease. | Credit: theasis via Getty ...

Sickle cell disease (SCD) is a genetic condition that affects ... This bowl shape is maintained by the healthy, blob-shaped hemoglobin protein. However, in SCD, mutations in hemoglobin change the ...