Base editing—the genome editing technique that alters base pairs using a nuclease-deficient Cas9 fused to a deaminase—was found to restart fetal hemoglobin expression in sickle cell disease (SCD) ...

(MEMPHIS, Tenn. – October 12, 2022) Scientists at St. Jude Children’s Research Hospital have shown how a protein responsible for adapting to low oxygen conditions (hypoxia), causes increased ...

Scientists at St. Jude Children's Research Hospital have shown how a protein responsible for adapting to low oxygen conditions (hypoxia), causes increased expression of fetal hemoglobin (HbF) in ...

Though the first CRISPR drug—Casgevy, the CRISPR-Cas9 therapy formerly known as exa-cel developed by Vertex Pharmaceuticals and CRISPR Therapeutics—was approved in the U.K. for the treatment of sickle ...

Researchers advancing gene-editing techniques to help patients with sickle cell disease discover an unexpected boost in fetal hemoglobin production, which mutes the effect of the disease. Help for ...

Sickle cell trait is where a person carries a single mutated hemoglobin A beta subunit allele. Usually, two mutated copies of this gene are needed to cause sickle cell disease (SCD). Typically, people ...

Sickle cell beta-thalassemia refers to an inherited condition that impacts hemoglobin. People with the condition have different changes in each copy of their hemoglobin gene. One causes red blood ...

HOUSTON - (June 3, 2019) - Help for patients with sickle cell disease may soon come from gene editing to fix the mutation that causes the disease and boost the patient's own protective fetal ...