The two serious blood diseases sickle cell anemia and thalassemia can soon be treated in Europe with the CRISPR-Cas technique. This is a genetic technique that cuts out a piece of DNA and can repair the genes of people who suffer from these blood diseases. The treatment technique was introduced last year in the United States and the United Kingdom. NOS reports this.
According to hematologist Erfan Nur of the Amsterdam UMC, not everyone with sickle cell anemia or thalassemia was eligible for the existing treatment. Nur calls the arrival of the CRISPR-Cas technology for these diseases a great development. But before people can actually be treated, the price still needs to be negotiated. The treatment probably costs around 2 million euros per patient. The price negotiations are expected to last about two years.
Body’s own material
The CRISPR-Cas technique is for patients aged 12 years and older and uses body’s own material. People treated with this will therefore hardly experience any side effects. Nevertheless, the treatment is quite intense, because doctors have to extract stem cells from the bone marrow so that the technology can pick out the gene defects in the lab. In the meantime, patients must undergo heavy chemotherapy so that the incorrect remaining stem cells also disappear. After the gene defects have been removed by CRISPR-Cas, the healthy stem cells are replaced.
Abnormal or too little blood
Patients with one of the two blood diseases have an abnormality in a gene that produces red blood cells. As a result, patients with sickle cell disease have abnormal blood and severe pain attacks. Patients with thalassemia have a shortage of red blood cells and must undergo many blood transfusions.
Also read: Ten patients with hereditary angioedema disease-free after CRISPR-Cas
By: National Healthcare Guide / Johanne Levinsky
2024-02-19 11:36:23
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