Forty-four-year-old Brian Madeux had is genes edited inside his body in early November, and in the process, made medical history.
Madeux, who suffers from Hunter Syndrome – a genetic disorder that causes joint stiffness, breathing problems and developmental delay – has had 26 surgeries to try to cope with his condition.
But now, he may never have to have another surgery.
The 44-year-old was hooked to IVs for three hours during a process that doctors believe will insert the gene he’s missing – the one that could end his Hunter Syndrome once and for all. If it works, the change will be permanent.
Sangamo Therapeutics, the company that performed the trial on Madeux, used microscopic gene-editing tools called zinc-finger nucleases to alter DNA inside Madeux’s body.
The reason this gene therapy is different (gene therapies have been performed since 1989) is its precision. Traditionally, gene therapy has relied on the insertion of a virus containing the right gene into the patient’s DNA – but the virus could put the gene in the wrong place, and it’s risky because those treatments have caused cancer in many cases.
“You know exactly where you’re going in the genome. It’s not like using a shotgun hoping you’re hitting a bird. It’s like using a rifle,” says Chester Whitley, a principal investigator on Sangamo’s clinical trial and a pediatrician at the University of Minnesota.
The treatment won’t reverse all the damage in Madeux’s body, but if it works, it could stop the disease from progressing any further.
Sangamo’s president, Sandy Macrae, says the ultimate goal is to cure children.
“Once we’re sure the medicine is safe and effective, our intention is to get it as quickly as possible into children,” Macrae said.
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