ISSN: 2041-6792

Clinical Investigation

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

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