Stem Cell Research and Regenerative Medicine

Genome Engineering Future

Genome engineering technologies based on synthetic nucleases and transcription factors allow for targeted gene sequence modification and expression. These engineered nucleases and transcription factors usually consist of a domain linked to an effector module that binds DNA. More recently, RNA-guided targeting of DNA sequences through the clustered regularly interspaced short palindromic repeats (CRISPR)/Cas system further simplified custom genome engineering, avoiding the need for the complex protein engineering needed for the ZFP- and TALE-based systems and thus enabling widespread use of genome engineering. Effector modules which can be attached to the targeted DNA-binding domains include catalytic endonuclease domains, transcription activators and transcription repressors.

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