"Fetal lung gene therapy - a once in a lifetime opportunity". With a title like that, who wouldn't be interested! The Ritchie Centre was very pleased to have Dr. Marcus Davey from the Children's Hospital in Philadelphia present some very exciting work about the possibility of treating diseases in the womb. As the Ritchie Centre is focused on fetal and neonatal health, this was one seminar that got a lot of attention.
Cystic fibrosis is a disease in which the lungs generate excess mucous that is thicker than normal and this can increase the patient's susceptibility to lung infections. Dr. Davey's research is using gene therapy to replace defective or missing genes in the fetus and therefore alleviate cystic fibrosis even before the baby is born. The gene therapy approach exploits the ability of viruses to promote the production of particular proteins and thus replace those that are defective/missing in cystic fibrosis. This research has been performed in a sheep model, in which viruses encoding functional proteins, are injected into the amniotic sac. The viruses are delivered right in front of the mouth of the fetus in the hope that the fetus inhales some of these viruses and the protein is then made in the lung cells. Indeed Dr Davey's research has shown that this technique works, however the number of lung cells with the ability to produce the new proteins is low. Now, the question is how can the delivery be maximised and what is the best way to delivery the viruses to the fetus? With such promising benefits to newborns, this is certainly an area of research we will be keeping our eyes on.
