Ibrahim Keru
Sickle cell disease is a genetic disease that affects the haemoglobin which is an essential protein in red blood cells causing the sickling of blood cells. This disease can be fatal and causes a lot of pain. A new life-changing treatment using CRISPR technology on sickle cell patients has been approved by the NHS.
Sickle cell disease
Sickle cell disease occurs because of a mutation in the haemoglobin protein that replaces the building blocks of the protein called glutamic acid with a different amino acid called valine creating abnormal beta globin chains. This mutation causes the red blood cells to have a sickle shape instead of a smooth biconcave shape which causes people that suffer from this disease intense pain. This disease mostly affects individuals from black African and Caribbean descent Approximately 15,000 people in England suffer from this disease. Individuals with this disease have a shorter lifespan and are at higher risk of anaemia, tissue and organ damage because the sickled red blood cells block blood vessels.
In January 2025, the NHS approved a one-off genetic treatment that uses Crispr gene editing technology to treat sickle cell. This treatment gives the people suffering from this hope and holds huge promise for a cure.
How the gene therapy works
This gene editing therapy called Casgevy is a step-by-step process. The blood stem cells, which is where all blood cells come from, are removed from the patient’s bone marrow first. Then Crispr the gene editing technology is used make precise edits on targeted genes. The Crispr technology works by leveraging the fact baby haemoglobin called foetal haemoglobin can’t be affected by sickle cell disease, so Crispr that targets the gene that make the adult haemoglobin turning them off. Patients will have to have tests to assess if their bodies are suitable for this treatment which involves a conditioning chemotherapy and then the altered stem cells will be transfused into their bodies where they will produce normal healthy red blood cells.
This treatment may have side effects such as bleeding and headaches and will require patients to stay in the hospital for an extended period of time after the treatment. This gene therapy will be available for patients older than 12 years old in NHS specialist centres in Birmingham, Manchester and England.
This treatment gives hope to the many people suffering from sickle cell disease in England that are unable to get a stem-cell transplant but can now get this CRISPR technology treatment. It also gives hope to the family and friends of people suffering from this disease. This treatment was previously approved in other countries and is now being administered to patients in other countries such as Germany and France and Casgevy gene-editing therapy has also been approved for another blood disorder called beta-thalassemia.
Find Out More!
https://www.imperial.nhs.uk/about-us/news/gene-editing-for-sickle-cell-approved-for-use-on-the-nhs
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Amazing article. I really enjoyed reading this and learning about sickle cell disease.
Very insightful. This article breaks down how this new treatment works very well.