title: It's all druggable.
abstract: 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.
mesh_terms: D000818:Animals; D000072669:Gene Editing; D020022:Genetic Predisposition to Disease; D016678:Genome / Q000187:drug effects*; D055106:Genome-Wide Association Study; D006801:Humans; D058990:Molecular Targeted Therapy; D011506:Proteins / Q000819:agonists / Q000037:antagonists & inhibitors / Q000235:genetics* / Q000378:metabolism; D054852:Small Molecule Libraries / Q000138:chemical synthesis / Q000627:therapeutic use*
chemical_list: D011506:Proteins; D054852:Small Molecule Libraries