US Edition3-Year-Old First to Receive Gene Therapy for Hunter Syndrome
Oliver Chu, the first patient to receive this gene therapy, has stopped weekly enzyme infusions and shows improved speech, mobility, and health nine months post-treatment.
- 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.
38 Articles
38 Articles
This Toddler Was the First Person to Receive a Test Treatment for a Rare Genetic Disorder. Nine Months Later, His Progress Is Inspiring Hope
Oliver Chu is one of five kids participating in a clinical trial investigating a gene therapy for Hunter syndrome, a disorder with symptoms akin to childhood dementia that limits life expectancy
A 3-year-old child became the first patient to receive an experimental genetic therapy to treat Hunter’s syndrome, a progressive hereditary disease affecting 1 in every 155,000 live births. The procedure was developed by researchers from Manchester University, England. The institution announced the medical advancement on Monday, November 24. The intervention was conducted at the Royal Manchester Children’s Hospital (RMCH) with support from the C…
World first: Three-year-old boy with rare condition defies odds after gene therapy
PRESS REVIEW – Tuesday, November 25: We look at reactions to an updated peace plan for Ukraine, which one paper says is an "acceptable end to the war." French President Emmanuel Macron is expected to unveil a voluntary military service plan for citizens. In Italy, three children removed from their parents after growing up off-grid in the Italian forest have sparked an intense discussion about alternative lifestyles. Plus, groundbreaking gene the…
A three-year-old boy is the first in the world to undergo gene therapy for the rare Hunter syndrome – and the results are promising. Just under a year after starting treatment, he is now developing normally. “It’s so amazing,” says his mother.
Oliver has an inherited condition called Hunter syndrome, which causes progressive damage to the body and brain.
At the University of Lisbon Sciences College, Diogo Fernandes is trying to develop a genetic therapy capable of rewriting the course of rare and incapacitating muscle disease.
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