It may take a lot longer than we think, but sooner or later
gene editing will change what medicine looks like. The biotechnology industry began in the 1970s when someone grafted
insulin into E. coli, showing that a human protein could be
manufactured outside the body. Now there’s a way to change
DNA where it lies, inside your genes. When he looked through
a microscope at his own cells in Olson’s lab, Dupree tried to
take the rational view: here was a solution for the next generation of boys. His mother, however, has allowed herself to hope.
“I was ecstatic. I remember thinking, ‘ This could be something
that works,’” Debbie says. Duchenne is a ticking clock. Parents
can’t help making the calculations: this long for animal studies, this many years for the first human trial, that much more
time until they know if it really works. Luckily, Ben’s disease is
the slow-moving kind. The doctors said he’d be gone by 19, but
he’s still here. And maybe he’ll still be here in 10 years, says his
mother, “so they can try it on him.”
Antonio Regalado is MIT Technology Review’s senior editor
“What I learned
about gene therapy
is that the rabbit does
not win the race.
The tortoise wins
Boys with muscular dystrophy demand action
on new drugs during a 2016 meeting at the U.S.
Food and Drug Administration.