Oliver Chu from California first person to have the one-off treatment for Hunter syndrome
Doctors are cautiously optimistic about a groundbreaking gene therapy for children affected by a devastating inherited disorder after seeing positive results in the first boy to receive the treatment.
Three-year-old Oliver Chu from California became the first patient to have the therapy nine months ago as part of a clinical trial run by researchers in Manchester. It is too early to call the therapy a success, but doctors are encouraged by his progress so far.
“Things look really hopeful right now, but Ollie was the first human to receive this therapy and it’s only been nine months out,” said Prof Simon Jones, consultant in paediatric inherited metabolic disease at the Manchester Centre for Genomic Medicine (MCGM) at Saint Mary’s hospital.
“We have four more boys scheduled in and we will need to prove the benefit is long-lasting,” said Jones, the joint leader of the trial.
