Gene Therapy For Inherited Disease In The Unborn Child

In the first part of this series , we explored how early genetic screening and gene therapy transform the lives of newborns and their families. Now, we’re taking an even earlier step: treating inherited diseases in the womb before birth. Until recently, even the most advanced gene therapies could only be given after a child was born—often racing against time to prevent irreversible damage. But what if we could intervene even earlier?

That’s the question now being answered, with some astonishing results. A recent study published in Science Translational Medicine showed that delivering a special kind of genetic therapy directly to the fetus could prevent the onset of spinal muscular atrophy in animal models. Treating the condition before birth may be possible to preserve healthy motor