STAT

Can gene therapy halt diseases in babies before they’re even born?

Imagine a virus being slipped into a pregnant woman’s womb, delivering a corrective gene to a fetus and remedying a disease before the baby is even born.

Gene therapies — in which a corrective gene hitches a ride on a virus into a patient’s cells — are being tested as potential cures or treatments for sickle cell, Duchenne muscular dystrophy, and a range of other diseases. Some patients have already been treated with a gene therapy for an inherited form of blindness, the first to be approved in the U.S.

Now, imagine the virus-gene rig slipping into a pregnant woman’s womb, where it would reach the fetus and start remedying a disease before the baby is even born.

That kind of procedure remains an untested goal for now — one whose feasibility has been debated by scientists for years. But advances in fetal gene therapies in animal

You’re reading a preview, subscribe to read more.

More from STAT

STAT2 min read
STAT+: Pharmalittle: We’re Reading About An Amgen Obesity Drug, A Senate Bill On Shortages, And More
Amgen will no longer develop an early-stage obesity pill, and will instead focus on a more advanced injectable candidate to compete with Wegovy and Zepbound.
STAT2 min read
STAT+: Pharmalittle: We’re Reading About AstraZeneca CEO Pay, Alternatives To WuXi And More
And so, another working week will soon draw to a close. Not a moment too soon, yes? This is, you may recall, our treasured signal to daydream about weekend plans.…
STAT2 min read
STAT+: Pharmalittle: We’re Reading About FTC Fighting ‘Junk’ Patents, Pfizer Direct-to-consumer Plans, And More
The FTC expanded its campaign against pharmaceutical companies for filing what it calls “junk” patent listings for 20 different brand-name treatments.

Related Books & Audiobooks