New Gene Therapy for the Treatment of Sickle Cell Disease: All You Need to Know [2025] скачать в хорошем качестве

New Gene Therapy for the Treatment of Sickle Cell Disease: All You Need to Know [2025]

A groundbreaking gene therapy for the treatment of sickle cell disease has been approved in England. Studies find that there is a ‘functional cure’ in 96.6 % of patients receiving this treatment. The approved gene therapy is known as exagamglogene autotemcel or exa-cel, which is also called Casgevy. Sickle cell disease is an inherited blood disorder. This condition is characterised by abnormal crescent or sickle shaped red blood cells. These sickled cells can get stuck in small blood vessels and block blood flow, causing pain or sickle cell crisis. Gene editing therapy involves collecting stem cells from a person. These cells are then edited in a laboratory to produce normal non-sickling red blood cells. The edited cells are then put back into the person. Exa-cel or Casgevy uses a person’s own edited cells. This means there is no risk of rejection by the body. It should be noted that this one-time gene editing therapy would be available for patients aged 12 and over with specific severe forms of sickle cell disease, and who are suitable for a stem cell transplant but without a matched donor. Exa-cel was approved in the United States in December 2023 for the treatment of sickle cell disease in patients aged 12 years and older. It is also available in European Union, Canada and Saudi Arabia. Gene therapy is being used for transfusion-dependent beta-thalassaemia and spinal muscular atrophy or SMA as well. It is hoped that gene therapy will be available to treat other genetic conditions such as cystic fibrosis and Duchenne muscular dystrophy. #gene therapy #sickle cell disease #anaemia #sickle cell crisis