Gene therapy halts deadly hereditary brain disease in two boys
Gene therapy has been used to treat two young boys with a devastating and fatal brain disease called adrenoleukodystrophy (ALD). Two years after treatment, both boys showed signs that the disease had stopped progressing and that there were no serious side effects from the gene therapy. These results, published in the journal Science, show huge promise, both for the future treatment of ALD and for the revival of investigations into the use of gene therapy to treat a wide variety ...