In Manchester, gene therapy has been used successfully for the first time in Hunter syndrome. This is a rare inherited disease that causes severe damage to the brain and internal organs. The first patient was three-year-old Oliver Chou from the United States. His defective gene was replaced in stem cells, after which the body began producing the missing enzyme and delivering it to the brain, something that had not been possible before.
Until now, treatment for Hunter syndrome consisted of lifelong weekly infusions of the enzyme, which could not stop the progressive decline in cognitive abilities. Now Oliver has not needed treatment for nine months, and his condition has improved noticeably — according to his parents and doctors, his speech, motor and cognitive skills have improved.
The study is in phase I, and the method will soon be tested on four more children from other countries. Similar work is already under way for other genetic diseases, and if effectiveness is confirmed, it could change the lives of thousands of children around the world. Gene therapy is gradually turning incurable diseases into manageable ones — and sometimes into curable ones.
