How do mutations happen, and how do they influence the future of a species? Molecules of sickle-cell hemoglobin stick to one another, forming rigid rods. These rods cause a person's red blood ...

The potential target, the FLT1 gene, contributes to the production of a protein, fetal hemoglobin, whose presence is already known to improve the lifespan of people with sickle cell disease.

Single gene disorders, like Huntington’s disease and cystic fibrosis, actually follow Mendelian inheritance patterns. The PKU-associated enzyme deficiency was determined biochemically in the ...

Today, CASGEVY is approved for use in patients with sickle cell disease in the United States and multiple countries in Europe and the Middle East. “It’s an amazing gift to have been able to ...