Researching Ways to Treat Disease Before Birth

The Galganski Lab’s goal is to develop new ways to treat disease before birth. The field of fetal medicine and surgery was founded on the premise that treating a fetus while they were still developing could halt or reverse disease that would severely impact them after birth. As prenatal diagnosis and fetal therapy has accelerated, anomalies such as congenital diaphragmatic hernia, myelomeningocele and twin to twin transfusion syndrome can now be safely treated before birth.

Concurrent to the field of fetal therapy, advances in gene delivery and gene editing have ushered in the reality of repairing errors in the genetic code. As a handful of gene therapies have hit the market recently for children and adults, the available prenatal treatments remain extremely limited. The onset of pathology for genetic diseases such as lysosomal storage disorders, hemoglobinopathies and skeletal dysplasias begins prenatally with devastating consequences soon after birth. Fetal gene therapy holds an opportunity to prevent the progression of disease. Furthermore, administering therapy prenatally may help develop tolerance to postnatal therapies due to the developing fetal immune system. To intervene successfully before birth could result in saving the lifetime of these affected children.