Approximately 100,000 people in the US have sickle-cell disease. Most sufferers are African-Americans, but there are also many Latino patients as well as people of Mediterranean, Middle Eastern, Asian, and Southeast Asian descent who have sickle-cell disease. The disease is painful, and shortens the lifespan of sufferers to about 40 to 60 years.
Although its cause has been understood for more than a century, patients with sickle-cell have historically been underserved by both the pharmaceutical industry and the medical establishment. However, as CRISPR is changing the face of medicine, it may also be changing this lived reality for people with sickle-cell disease, which is caused by a single mutation that is well-studied, making it an appealing candidate for correction with the gene-editing tool.
