Sickle cell diseases arises from hemoglobin protein mutations. The mutation causes red blood cells to form a sickle shape rather than a round one. One study suggests that this mutation could be ...

A drug from Fulcrum Therapeutics showed promise in a sickle cell study, raising the possibility that a pill could be an ...

It’s a reality that patients with sickle cell disease can only wish for: Walk into a clinic, get a shot and, soon after, be free of disease. No desperate search for a stem cell donor.

Hemoglobin normally is contained within blood cells -- it is what makes them red -- and that severely limits its ability to react with nitric oxide. The misshapen blood cells of sickle cell ...

On the heels of the U.K.'s authorizing the world’s first gene-edited treatment for sickle cell disease on Nov. 16, the U.S. Food and Drug Administration (FDA) approved the same treatment on Dec. 8.

In patients with sickle cell disease, there's a single, small mutation in the gene that encodes hemoglobin. The mutation is a switch of a single nucleotide, or base, (often represented by letters ...

In the case of sickle cell, the mutations in the gene coding for hemoglobin can cause blood cells to form a sickle shape, rather than a spherical one, and clog up small blood vessels, leading to ...

Rifkin-Zenenberg said the gene therapies are intended for patients with severe Sickle Cell Disease, particularly those with hemoglobin SS disease —the most severe form, affecting about 65% of ...