A one-year-old girl diagnosed with incurable aggressive leukemia is now in remission after receiving “designer cells” from a donor. The therapy made use of a powerful new gene-editing technique that could eventually be used to treat an array of hereditary diseases.
For 26 years, since researchers first identified the gene responsible for cystic fibrosis, scientists have attempted to use the knowledge to help treat the illness. Now, gene therapy has been shown to help sufferers for the first time.
A team of surgeons in Oxford have used a pioneering new form of gene therapy to stop six of their patients going blind--and it's hoped the technique could be used to treat blindness more generally.