It is evident from a recent market survey on gene editing in drug discovery that CRISPR/Cas9 is now recognised as the superlative method when attempting a gene knockout or when introducing defined mutations, insertions or modifications to the genome.
The development of genome editing technology is revolutionising the study of gene function and has the potential to usher in a new class of therapeutics for a broad range of diseases.
The recent high-profile translational failures in mouse models have highlighted the need for more relevant animal models. Advances in gene editing tools, including the CRISPR/Cas9 system, have enabled the modification of highly translational organisms such as rats and rabbits, and have also greatly reduced model development timelines.
We are in the midst of a revolution in genome engineering, based on reagents that can be designed to cut chromosomal DNA at arbitrary sites. These targetable nucleases allow creation of new mutations at specific sites, introduction of designed sequence changes and production of larger alterations, as desired by the experimenter. They are being used to explore gene function and create disease models and they hold great promise for human gene therapy.
It is now readily accepted that we are in a post genomic era. With the steady flow of genomic information available to researchers worldwide, the focus turns to ways to analyse this information effectively and then utilise it in a practical manner.