Results from clinical trials released today indicate that 2 patients, 1 with beta-thalassemia and the other with sickle cell disease, have potentially been cured
Results from clinical trials released Tuesday indicate that two patients, one with beta thalassemia and one with sickle cell disease, have potentially been cured of their diseases. The two trials, which involved using Crispr to edit the genes of the patients in question, were jointly conducted by Vertex Pharmaceuticals and CRISPR Therapeutics.
CTX001, the gene-editing therapy used in these trials, is “very surgical in how it makes the change,” says David Altshuler, Vertex’s chief scientific officer.
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