Currently, gene therapy for sickle cell disease is only available as part of clinical trials. That means that the full risks and benefits of the treatment have not been assessed in a large number ...

Some doctors told him he'd live with the disease forever, but Khaled Alsheebani never stopped believing treatments would ...

He first had sickle cell symptoms at age 2, and his symptoms were only getting worse as he got older. In this method, the researchers removed blood stem cells from Branden, which were then exposed to ...

Repositioning genes awakens fetal hemoglobin to treat disease. CRISPR editing may change future gene therapy. Researchers ...

Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them ...

When available, the educational toolkit will provide patients with sickle cell disease information on the gene therapy ...

Advocates say a "Saturday Night Live" skit about new sickle cell gene therapies perpetuated falsehoods that have ... explains, “I’m just going to swap this out for a Boogie Woogie Santa ...

New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living with the condition. Skip to Article Set weather ...

Aaron Scott, of Fayetteville, is the first in North Carolina to receive a new FDA-approved therapy which uses the patient's own cells to relieve the pain of sickle cell disease.

That troubles Victoria Gray of Forest, Miss., the woman who was the first person with sickle cell to get the gene-editing treatment. NPR broke the news when Gray got treated in 2019.

Hoffman M. FDA approves bluebird bio’s lovo-cel gene therapy for sickle cell disease. CGTLive™. December 8, 2023. Accessed October 16, 2024.

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.