Sickle cell anaemia (SCA) is a genetic blood disorder ... This leads to anaemia SCA is caused by the mutation of HBB gene. The HBB gene is responsible for the production of beta-globin, an ...

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

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

We can do better. We can build a system that provides coordinated high-quality care to every patient who needs it.

Since two groundbreaking treatments were approved in late 2023, only a handful of patients have been able to access the ...

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

One year on from the landmark U.S. approval of two powerfully effective gene therapies for sickle cell disease, the treatments have been barely used, a sluggish start that reflects the myriad ...

Sickle cell is a disease that causes red blood cells ... It is caused by a mutation in the hemoglobin gene (HBB). CRISPR-Cas9 acts as molecular scissors that can precisely snip out parts of ...

Research from UAB published in the New England Journal of Medicine in February 2022 suggests a gene therapy called LentiGlobin could provide a drastic improvement in quality of life for people with ...

The influential Institute for Clinical and Economic Review (ICER) in the US has issued draft guidance suggesting that new gene therapies for sickle cell disease (SCD) from Vertex Pharma and ...