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 ...

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 ...

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 ...

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 ...

The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 gene-editing technology in humans to directly correct the genetic mutation that causes the disease. Sickle cell disease is a genetic ...

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 ...

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