Synopsis

This report on Emerging Gene expression regulation technologies in medical (red) biotechnology contains the conclusions of a scientific literature evaluation carried out independently by Xendo commissioned by RIVM GMO office. The report focusses on the novel molecular genetic techniques with a medical application in order to ultimately affect disease related gene expression. The major genetic engineering technology areas that have been identified are: genome and epigenome editing, gene expression regulation and gene delivery. The technologies identified are ZNF (Engineered nuclease), TALENs (Engineered nuclease), CRISPR/Cas9 (Engineered nuclease system), siRNA and miRNA, and Antisense Oligonucleotides (ASOs). Moreover, advances in both viral and nonviral delivery systems are introduced as a general driver for the described genetic engineering technologies.