A World First: Gene therapy offers Hope for Hunter Syndrome
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A medical feat and a world first.This is the story of little Oliver Chu, a three-year-old boy affected by a rare hereditary disease called Hunter syndrome, or MPSII. This condition causes progressive damage to the body and brain. Due to a defective gene, Oliver was unable to produce an enzyme essential for maintaining cell health. This pathology affects approximately one boy in 100,000. Tragically, in Oliver’s family, his brother is also affected by the same pathology.
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Until now, there was only one drug available for Hunter syndrome but its cost was a staggering £300,000 per patient per year. This medication helps slow the progression of the physical symptoms of the disease but is ineffective in combating cognitive disorders.
Pioneering Gene Therapy offers Hope to Young Oliver
Oliver is undergoing a groundbreaking treatment involving stem cell modification and re-injection, offering a potential cure for his condition. His blood is processed through a specialized machine that isolates stem cells, as explained by Dr. Claire horgan, consultant pediatric hematologist, to the BBC.
These collected stem cells are then sent to a laboratory where scientists, led by Dr. Karen Buckland from GOSH’s cell and gene therapy department and principal investigator at UCL, utilize a viral vector to insert a functional copy of the defective gene into each cell. The hope is that once these modified cells are reintroduced into Oliver’s body, they will repopulate his bone marrow and begin producing healthy white blood cells.This innovative approach represents a significant step forward in gene therapy and offers a beacon of hope for Oliver and others facing similar challenges.
Gene Therapy Shows Promise for Rare Genetic Disorder
Oliver’s parents, Jingru and Ricky, hope this innovative therapy will one day be available to Skyler, oliver’s older brother. For his part, Professor Ricky is delighted: “He is doing really well.We observed progress in his language and mobility. In just three months, he has matured.” The very good news is also that Oliver was able to stop the weekly infusion of the missing enzyme. “I find it hard to believe it every time I tell people that Oliver makes his own enzymes. […] Every time we talk about it, I want to cry because it’s so amazing.”