Artificial Endonucleases for Genome Editing
Genome editing refers to methods for altering a specific gene in the chromosome of a living cell. The alteration might entail deletion or replacement with a different DNA molecule that is introduced to the cell, among other possibilities. Methods have been developed that enable researchers to alter a specific gene and test the effect of the alteration on the organism and, for gene therapy applications, to cure disease by correcting a defective gene.
Current genome editing methods involve generating a double-strand break at a specific site in the genome, followed by repair of the break by cellular DNA repair processes. Double-strand breaks can be introduced by an engineered zinc finger nuclease (ZFN) or a TAL effector endonuclease (TALEN). More recently, the CRISPR/Cas9 system has been used to generate RNA-directed DNA breaks.