Katie was diagnosed with Batten disease in 2011 and is now completely dependent on her parents. A visit to a neurologist raised the improbable possibility of Batten disease; months of trips to Starship Hospital ultimately confirmed it. Modified viruses carrying a corrective copy of the mutated Batten disease gene are injected into the fluid filled spaces in the brain, before "infecting" the brain cells and using them to make functional protein, which can correct or stabilise the disease. Another trial targeted at sheep with early symptoms has showed the disease stabilising the disease for more than a year, halting further decline. "Given early enough, I think gene therapy has a real chance of preventing this disease," Dr Mitchell said.

Source: Otago Daily Times September 10, 2017 21:33 UTC