Around 2,500 people are living with sickle cell disease in the Bay State, and they are advocating for a bill that would improve quality of life for all those dealing with the disease.

About Sickle Cell Disease (SCD) Sickle cell disease, a severe inherited blood disease, is caused by a single point mutation, E6V, in the beta globin gene.

The decision has ushered in a new era for sickle cell disease treatment — and marked the world’s first approval of a medicine based on CRISPR/Cas9 gene-editing technology.

In the UK, approximately 17,500 people have sickle cell disease and 300 babies are born with the condition each year. It is a genetic disorder caused by inherited mutations in a person’s DNA ...

It is a CRISPR-based medicine for sickle cell disease that partially silences BCL11A to boost the production of fetal hemoglobin, which is made with γ-globin.

Learn about sickle cell disease, its impact on the Black community, and the importance of genetic counseling. Know your status.

UCSF Benioff Children’s Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 ...

Sickle cell disease is a genetic blood disorder caused by a single mutation in both copies of a gene coding for beta-globin. Beta-globin forms half of the oxygen-carrying molecule hemoglobin. This ...

A person inherits sickle cell disease when they receive two sickle cell genes, one from each parent. Learn who can inherit this condition and how it passes from parents to children.

Given the extreme neglect of sickle cell disease to date, the sheer number of people affected and all we could learn by studying the illness, we urgently need a global initiative to advance ...

The treatment you receive for sickle cell disease will depend on your specific symptoms. Possible treatments include blood transfusions, medications such as hydroxyurea, and emerging therapies.

To safely target BCL11A for sickle cell disease, the team members identified a region within the gene that’s crucial for BCL11A expression specifically in red blood cells.