FOR THE first time ever, doctors have successfully used gene therapy to treat children with a rare disease of the nervous system.
The European Association for Leukodystrophy (ELA) said the breakthrough was unveiled by French researchers at a congress in Rotterdam of the European Society of Gene and Cell Therapy. The therapy targeted faulty genes that cause adrenoloeukodystrophy (ALD), a disease which gained worldwide recognition thanks to the 1992 film Lorenzo’s Oil with Susan Sarandon and Nick Nolte.
