Targeted Mutagenesis, Mouse

Genome-wide association screens (GWASs) for a large number of human genetic diseases generate novel hypotheses about familial and sporadic mutations. Gene targeting in embryonic stem cell (ESC), along with the new repertoire of zinc-finger nucleases (ZFNs), TAL effector nucleases (TALENs) and recombinase-mediated cassette exchange (RMCE) techniques, the increasing number of null, point, conditional, large deletions, inducible, reporter, hypomorphic alleles, combined with additional breeding schemes, different mutant backgrounds and several reporter lines, represent a vast repertoire of molecular tools that provides everything needed to target essentially any gene of interest in virtually any cell type, making imagination the only limiting factor. These techniques will allow us to investigate novel hypotheses in model organisms, which are no longer limited to the mouse. Correction of inherited mutations in adult human stem cells and even zygotes may be done with great efficiency in the future.