2018 preview: Gene therapy treats disease while in the womb

fetus

Dr G. Moscoso/Science Photo Library

“You can see during pregnancy that they already have bone fractures in the womb,” says Cecilia Götherström of the Karolinska Institute in Stockholm, Sweden. She is head of what will be the world’s first ever trial of giving fetuses stem cell therapy in the womb.

The aim is to relieve symptoms of, or even cure, osteogenesis imperfecta, known as brittle bone disease. Babies born with this rare condition have bones that fracture easily, caused by having faulty genes for collagen, the protein that normally reinforces and strengthens bones.

Götherström hopes to prevent this before babies are even born, by injecting them with healthy stem cells that have been extracted from donated tissue from aborted fetuses.

The team will specifically inject mesenchymal stem cells, which should go on to make bone with fully functioning collagen. “We hope they will home to bones,