
People with Sickel-Cell Anemia can have their disease cured through the use of CRISPR, a method of altering DNA. The technique involves intensive treatment that right now makes CRISPR very expensive and very difficult for the patient, but, for sickle cell patients, it might be worth the cost, the pain, and the time.
The Age of Crispr Medicine Is Here – www.wired.com
Excerpt:
So far, only nine centers across the US are currently offering Casgevy, which may limit who gets access to it. Vertex says the number of participating sites will grow in the coming weeks and months…
Collecting stem cells from the blood can take hours, and multiple sessions may be needed to get enough cells to edit. After that is a harsh conditioning regimen. Patients must undergo chemotherapy to kill any lingering diseased cells and make room in the bone marrow for the newly edited ones. Chemotherapy can cause mouth sores, fatigue, hair loss, nausea, and other unpleasant side effects. It can also result in infertility. Vertex also plans to offer fertility support to commercially insured patients, but the benefit won’t extend to Medicaid recipients. In the US, freezing eggs and sperm can cost thousands of dollars, not to mention the cost of IVF.
Patients also need to be hospitalized for weeks while the edited cells make their way to the bone marrow and start making new blood cells. Olaghere spent a total of 17 weeks in the hospital to get Casgevy.
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