Wikimedia CommonsA 12-year-old US boy became the first patient treated for, and hopefully cured of, sickle-cell anemia since a new gene therapy was approved. The genetic disease, which causes misshapen red blood cells, leads to excruciating pain, damaged organs, and shortened lives. The therapy involves removing bone marrow, editing the cells, and reimplanting them. It was, reported The New York Times, an arduous process: Kendric Cromer was in hospital for 44 days, with his existing marrow destroyed by chemotherapy, leaving him in agony. Other patients who underwent the new therapy refused to talk about it. But it worked for them and is believed to have worked for Kendric. Gene therapies are changing other diseases too: Cystic fibrosis, once a death sentence, could become curable. |