Envisioning treating genetically-defined urinary tract malformations with viral vector mediated gene therapy – Commentary

Abstract

I suspect that many pediatric urologists are already familiar with the ground-breaking genetic therapies available for devastating monogeneic conditions such as spinal muscular atrophy [1,2]. These expensive yet effective therapies have certainly made waves in both the scientific literature and lay press. However, I wonder how many pediatric urologists have stopped to consider whether such gene therapy could be applied to our own specialty. Admittedly, as I read about such therapies for SMA, I did not consider that this technology could be applied to urology.