Key Points
The cells clumped together, blocking blood flow and unleashing excruciating bouts of pain..
The disease can be cured with a bone marrow transplant, which involves replacing a patients stem cells with healthy ones from a donor so that the patient can make normal red blood cells..
Making a cut in the gene releases the brakes, allowing cells to make the fetal type and override the abnormal adult kind.. In 1948, pediatrician Janet Watson noticed that children with sickle cell disease had normal blood cells as infants, but that the cells became sickled around six months..
Then the group identified just a portion of the gene that could be inactivated and still turn on fetal hemoglobina safer approach, since genes often have many functions. In 2015, they showed that this stretch of DNA could be edited out with Crispr to boost fetal hemoglobin in mice and human cells...
I know there are going to be many patients who dont go down this road because of fertility issues and the need to be in the hospital for a while, says Sharl Azar, medical director of the Comprehensive Sickle Cell Disease Treatment Center at Massachusetts General Hospital, one of the initial centers to offer Casgevy..
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