Oliver's Miracle: Hope, Gene Therapy 💖🎉

World News

Oliver Chu, a three-year-old boy, is the first person with Hunter syndrome to receive gene therapy. Hunter syndrome, also known as MPSII, is a rare, inherited condition causing progressive damage to the body and brain, often leading to premature death. Oliver was unable to produce the enzyme iduronate-2-sulfatase (IDS) due to a genetic error. In a world first, medical staff in Manchester attempted to halt the disease by altering Oliver’s cells using gene therapy. Oliver was the first of five boys worldwide to receive the treatment. A specific type of cell called stem cells were collected from Oliver and sent to Great Ormond Street Hospital (GOSH) in London, where scientists inserted the missing IDS gene into a virus. The treatment aims to correct the genetic error and halt the accumulation of large sugar molecules in tissues and organs. On February 2025, Oliver, a child with Hunter syndrome, received a gene therapy infusion at Great Ormond Street Hospital (GOSH). The treatment involved inserting a working copy of the faulty gene into stem cells, which were then intended to repopulate his bone marrow and produce the missing enzyme. To facilitate enzyme passage into the brain, the inserted gene was modified. Following the initial infusion, a second identical infusion was administered on the same day. In May 2025, Oliver returned to Royal Manchester Children's Hospital for follow-up tests, showing improved mobility, speech, and engagement, and was able to discontinue the weekly enzyme infusion. The family, including Oliver, Jingru, Ricky, and Skyler, returned to California. Five boys, from the US, Europe, and Australia, were enrolled in a sickle cell gene therapy trial. Skyler, a boy with delayed development in speech and motor skills, was initially considered too old for the trial, but tests showed he was still largely unaffected. Oliver, Ricky’s younger brother, received the treatment and has thrived, nine months post-transplant, exhibiting significant improvements in speech, agility, and cognitive development, producing hundreds of times the normal amount of enzyme. Follow-up tests occur every three months. The trial aims to monitor the boys for at least two years. If successful, the hospital and university intend to partner with a biotech firm to license the gene therapy approach, which is also being applied to other gene disorders.