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In 2023, the first drug making use of CRISPR gene editing, Casgevy, was approved for use in the United Kingdom to cure sickle-cell disease and beta thalassemia. [ 13 ] [ 14 ] Casgevy was approved for use in the United States on December 8, 2023, by the Food and Drug Administration .
Sickle cell trait causes a change in the haemoglobin molecule in the blood. Normally, red blood cells have a very flexible, biconcave shape that allows them to move through narrow capillaries ; however, when the modified haemoglobin S molecules are exposed to low amounts of oxygen, or crowd together due to dehydration, they can stick together ...
Sickle-cell versions of hemoglobin stick to themselves, stacking to form fibers that distort the shape of red blood cells carrying the protein. These sickle-shaped cells no longer flow smoothly through blood vessels, having a tendency to clog or degrade, causing the medical problems associated with this disease. [citation needed]
Sickle-cell anemia, which can cause blood cells to clump up and block blood vessels, can also lead to stroke. Stroke is the second leading cause of death in people under 20 with sickle-cell anemia. [52] Air pollution may also increase stroke risk. [53]
However, in systemic lupus erythematosus it is 4 to 14%, [106] and in sickle cell disease, it ranges from 20 to 40%. [107] Up to 4% of people who develop a pulmonary embolism go on to develop chronic thromboembolic disease including pulmonary hypertension. [ 40 ]
Centrosome. Cell membrane. The cell nucleus (from Latin nucleus or nuculeus 'kernel, seed'; pl.: nuclei) is a membrane-bound organelle found in eukaryotic cells. Eukaryotic cells usually have a single nucleus, but a few cell types, such as mammalian red blood cells, have no nuclei, and a few others including osteoclasts have many.
Blood transfusion is the process of transferring blood products into a person's circulation intravenously. [1] Transfusions are used for various medical conditions to replace lost components of the blood. Early transfusions used whole blood, but modern medical practice commonly uses only components of the blood, such as red blood cells, plasma ...
Multiple sclerosis. Variable, including almost any neurological symptom or sign, with autonomic, visual, motor, and sensory problems being the most common. [1] Multiple sclerosis (MS) is an autoimmune disease in which the insulating covers of nerve cells in the brain and spinal cord are damaged. [3] Being a demyelinating disease, MS disrupts ...