Base editing utilizes enzymes to modify single amino acids at the most foundational unit of DNA, called a base.

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 ...

Abnormal cells can clog blood vessels, leading to severe pain episodes and restricting oxygen delivery to vital organs ...

Immvention Therapeutix is partnering with Novo Nordisk to develop oral treatments for sickle cell disease (SCD) and other ...

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 ...

Sickle-cell anemia is due to a mutation in the gene encoding the β-subunit of hemoglobin (called HBB), which leads to an abnormal structure of the β-subunit protein chain and the sickle-cell ...

A groundbreaking study reveals that individuals with sickle cell disease show premature brain aging, appearing 14 years older ...

Individuals with sickle cell disease – a chronic illness where misshapen, sticky blood cells clump together, reducing oxygen ...

DNA contains genes that carry instructions for the manufacture of a protein. If a mutation occurs ... they will have the genetic condition sickle cell anaemia. The sickle shaped red blood cells ...

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 ...

New data from a Phase 1b clinical trial testing Fulcrum's pociredir for sickle cell disease are expected by the middle and ...