Breakthrough in Hunter Syndrome Treatment

Oliver Chu, 3, first global recipient of gene therapy for Hunter syndrome.
University of Manchester researchers led by Prof Brian Bigger and Prof Jones developed the therapy.
Modified IDS enzyme gene delivered via 125 million stem cells in two doses.
Oliver shows improved speech, mobility, and cognition post-treatment.
University handed back license; LifeArc grant enabled five international trial participants.
Treatment requires three-monthly and two-year monitoring.

Medical Breakthrough | Gene Therapy | Clinical Trial

United States | Australia | Europe

Oliver Chu | Prof Brian Bigger | Prof Jones | LifeArc

1 month ago