In a pioneering clinical trial run by CRISPR Therapeutics and Vertex Pharmaceuticals, a single infusion of genetically modified cells has eliminated severe symptoms of sickle cell in one woman with the disorder. The cells were modified by using the gene-editing tool CRISPR to switch on a gene in the patients’ blood stem cells so that they could make healthy hemoglobin.
To do this, doctors first remove these cells from the body then send them off to a manufacturing facility to be edited with CRISPR. After that, scientists perform several safety tests on the cells, freeze them, then transport them back to the hospital. Once thawed, the cells are infused back into the person, where they can grow into healthy new blood cells.
That therapy also involves removing a patient’s blood stem cells and genetically modifying them. But instead of editing the cells, scientists use a technique that’s older than CRISPR.
Name of Author: Emily Mullin