Advances in targeted genome editing

► Genome editing is the modification of genes, typically by engineered nucleases. ► Advances in targeted DNA-binding proteins and nuclease activity have accelerated progress. ► Novel studies of genes and proteins have resulted from altering genome sequences. ► New animal models have been developed for several species. ► Genome editing is in preclinical studies and clinical trials for several diseases. New technologies have recently emerged that enable targeted editing of genomes in diverse systems. This includes precise manipulation of gene sequences in their natural chromosomal context and addition of transgenes to specific genomic loci. This progress has been facilitated by advances in engineering targeted nucleases with programmable, site-specific DNA-binding domains, including zinc finger proteins and transcription activator-like effectors (TALEs). Recent improvements have enhanced nuclease performance, accelerated nuclease assembly, and lowered the cost of genome editing. These advances are driving new approaches to many areas of biotechnology, including biopharmaceutical production, agriculture, creation of transgenic organisms and cell lines, and studies of genome structure, regulation, and function. Genome editing is also being investigated in preclinical and clinical gene therapies for many diseases.