It's all druggable

Current medicines are small chemicals that target the activity of proteins in the body, resulting in therapeutic and off-target changes in metabolism and gene expression. Now, however, gene expression levels can be raised or lowered at almost any point in the genome by sequence-targeted therapeutics, and the effects of these agents can be tested in preclinical models generated by gene editing of experimental animals and human cells. Genome-wide association studies (GWAS) of disease predisposition, metabolism and gene expression have a key role in explaining how current protein-targeting drugs work and in using the regulatory variation in human genomes to guide the therapeutic future of targeted gene regulation.