ABOVE: The therapy targets a gene in the liver.
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The first patients to have received a medium dose of a gene therapy based on in vivo gene editing have lower levels of sugar in their urine—a hopeful sign that the intervention for Hunter syndrome is working. However, clinicians did not observe an increase in levels of the enzyme they hoped to replace, Sangamo Therapeutics, which developed the treatment, reported today (September 5).
“I cannot absolutely say it’s a treatment effect,” study leader Joseph Muenzer of the University of North Carolina, Chapel Hill, tells the Associated Press. But he adds that the data are “really encouraging.”
Hunter syndrome is a rare disease in which an enzyme deficiency leads to a buildup of complex polysaccharides. Those with the condition often die young.
The first person to have received the treatment got it back in November. The infusion includes a normally ...
