Doctors say they constructed a bespoke gene-editing treatment in less than seven months and used it to treat a baby with a deadly metabolic condition. The rapid-fire attempt to rewrite the child’s DNA marks the first time gene editing has been tailored to treat a single individual.
The baby who was treated, Kyle “KJ” Muldoon Jr., suffers from a rare metabolic condition caused by a particularly unusual gene misspelling. Researchers say their attempt to correct the error demonstrates the high level of precision new types of gene editors offer.
The project also highlights what some experts are calling a growing crisis in gene-editing technology. That’s because even though the technology could cure thousands of genetic conditions, most are so rare that companies could never recoup the costs of developing a treatment for them. Read the full story.
—Antonio Regalado
Access to experimental medical treatments is expanding across the US
—Jessica Hamzelou
A couple of weeks ago I was in Washington, DC, for a gathering of scientists, policymakers, and longevity enthusiasts. They had come together to discuss ways to speed along the development of drugs and other treatments that might extend the human lifespan.
One approach that came up was to simply make experimental drugs more easily accessible. Now, the state of Montana has passed a new bill that sets out exactly how clinics can sell experimental, unproven treatments in the state to anyone who wants them.
The passing of the bill could make Montana something of a US hub for experimental treatments. But it represents a wider trend: the creep of Right to Try across the US. And a potentially dangerous departure from evidence-based medicine. Read the full story.
