LONDON, March 15, 2024 – A patient in the United Kingdom has become the first person to cease regular blood transfusions thanks to a groundbreaking gene-editing therapy, offering a potential cure for severe sickle cell disease and beta thalassemia.
Gene Editing Offers Hope for Blood disorder Patients
A new therapy is allowing a patient to live transfusion-free, marking a important step forward in treating inherited blood disorders.
- The gene-editing therapy, known as Casgevy, modifies the patient’s own blood stem cells.
- The patient, who has beta thalassemia, had relied on regular transfusions for years.
- This success represents a major milestone in the field of gene therapy.
- The treatment is currently available through the National Health Service (NHS) in England.
For individuals battling debilitating inherited blood disorders, the prospect of a life free from frequent blood transfusions has long been a distant dream. Now, that dream is becoming a reality for at least one patient in the UK, thanks to a revolutionary gene-editing treatment. This breakthrough offers a beacon of hope for thousands living with conditions like sickle cell disease and beta thalassemia.
How Does the Gene-Editing Therapy Work?
The therapy, called Casgevy, utilizes CRISPR-Cas9 technology to edit the patient’s own blood stem cells. These modified cells are then reintroduced into the body, prompting the production of healthy hemoglobin. Hemoglobin is the protein in red blood cells that carries oxygen throughout the body. The process essentially reactivates fetal hemoglobin, a form of the protein typically only produced in the womb, which can compensate for the defective hemoglobin in these disorders.
The patient,who has not been publicly named,suffered from beta thalassemia,a genetic blood disorder that reduces the production of hemoglobin. Prior to the treatment,
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