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3-Year-Old First to Receive Gene Therapy for Hunter Syndrome
Oliver Chu, the first patient in a global trial, stopped weekly enzyme infusions and shows improved speech and mobility nine months after gene therapy, researchers say.
- In recent months at Royal Manchester Children's Hospital, three-year-old patient Oliver Chu became the first in the world to receive gene therapy for Hunter syndrome, now producing the missing enzyme.
- After more than 15 years of research, University of Manchester researchers led by Prof Brian Bigger and Prof Jones developed a gene therapy inserting a modified IDS enzyme gene to cross the blood–brain barrier.
- After modification at GOSH, the cells were infused back into Oliver as around 125 million gene‑modified stem cells in two short doses of about 10 minutes each.
- In follow up, doctors note Oliver Chu shows gains in speech, mobility, cognition, has stopped weekly enzyme infusions, is nine months post treatment, and follows a three-monthly and two-year monitoring schedule.
- Looking to the future, the university handed back the licence, and LifeArc's £2.5m grant rescued the trial, enabling five boys from US, Europe, and Australia to enroll, with licensing prospects if successful.
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Total News Sources29
Leaning Left2Leaning Right6Center5Last UpdatedBias Distribution46% Right
Bias Distribution
- 46% of the sources lean Right
46% Right
15%
C 39%
R 46%
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