This is a guest post from Mari Niemi at the Wellcome Trust Sanger Institute. Mari is a graduate researcher whose research combines the results of human genetic studies with zebrafish models to study human disease.
The turn of the year 2012/13 saw the emergence of a new and exciting – and some may even say revolutionary – technique for targeted genome engineering, namely the clustered regularly interspaced short palindromic repeat (CRISPR)-system. Harboured with the cells of many bacteria and archaea, in the wild CRISPRs act as an adaptive immune defence system chopping up foreign DNA. However, they are now being harnessed for genetic engineering in several species, most notably in human cell lines and the model animals mouse (Mus musculus) and zebrafish (Danio rerio). This rapid genome editing is letting us to study the function of genes and mutations and may even help improve the treatment of genetic diseases. But what makes this technology better than what came before, what are its downsides, and how revolutionary will it really be?
Genetic engineering – then and now
Taking a step backward, the ability to edit specific parts of an organism’s genetic material is certainly not novel practice. In the last decade or two, zinc finger nucleases (ZFNs) and more recently employed transcription activator-like endonucleases (TALENs) saw the deletion and introduction of genetic material, from larger segments of DNA to single base-pair point mutations, at desired sites become reality. ZFNs and TALENs are now fairly established methods, yet constructing these components and applying them in the laboratory can be extremely tedious and time-consuming due to the complex ways in which they binding with DNA. Clearly, there is much room for improvement and a desire for faster, cheaper and more efficient techniques in the prospect of applying genome engineering in treatment of human disease.