In June last year, Helen Obando, lay in her hospital bed waiting for a bag of stem cells from her bone marrow, modified by gene therapy, to start dripping into her chest. She was admitted to Boston’s Children’s Hospital for the treatment of Sickle cell disease, an inherited blood disorder that can cause excruciating pain, organ damage and even early death.
Scientists have been experimenting with the gene therapy for two decades now. And it will be years before they know if this new procedure, which aims to flip a genetic switch so the body produces healthy blood, is effective in the long run. If it is, sickle cell disease could be the first common genetic disorder to be cured by manipulating human DNA.