Abstract

With advances in techniques like CRISPR gene editing and the use of viral vectors, the number of gene therapy trials has rapidly grown in recent years, promising to deliver solutions to diseases that were previously considered untreatable.

There are however several challenges facing the developers of these therapies, including low vector concentration, empty capsids, contaminating host cell DNA, contamination by mycoplasmas and the common thread amongst these is the need for rigorous quality control. Here we will look in more detail at how ddPCR can be used to overcome some of these challenges and help guarantee safe and effective gene therapies.