FDA Approves CRISPR Treatment for Sickle Cell

The Food and Drug Administration (FDA) has approved a gene-editing tool called Casgevy for treating patients with certain blood disorders, including sickle-cell disease. Casgevy utilizes CRISPR technology to edit the DNA of a patient’s own adult stem cells. In the case of sickle cell disease, the therapy aims to address a mutation that affects the ability of red blood cells to carry oxygen. Casgevy makes a cut to turn off an inhibitor, allowing the production of fetal hemoglobin to replace the mutated adult hemoglobin.

The discussion also touches on bioethical considerations, distinguishing between somatic genetic engineering (on already born individuals) and germline or heritable genetic engineering (on embryos). The latter raises ethical concerns about potential long-term effects on future generations. The interview emphasizes the importance of maintaining high ethical standards in gene-editing research and avoiding the pursuit of technologies that cross ethical boundaries.