Abstract
Over the past decade, research into the biology and applications of CRISPR-Cas systems has become one of the most dynamic and evolving fields in the life sciences, with great promise for future biotechnical and biomedical developments.
CRISPR-Cas is originally an RNA-mediated immune system that has evolved extensively in bacteria and archaea into multiple systems and subsystems and whose function is to protect these microorganisms from invasion by mobile genetic elements such as viruses (phages). Decoding the detailed mechanism of one of the CRISPR-Cas systems - CRISPR-Cas9 – with my team has led to the development of a transformative technology in the biological sciences, enabling rapid and efficient targeted genome editing, chromosome tagging and gene regulation in a wide variety of cells and organisms. The RNA-programmable CRISPR-Cas9 tool is efficient, versatile, easily programmable, and has been widely adopted by the scientific community.
For a decade now, the CRISPR-Cas9 system has been an integral part of every researcher's toolbox for manipulating genetic information by introducing or correcting mutations, replacing genes, modifying DNA or modulating transcription in a cell or organism in a targeted manner - and applications of this revolutionary technology continue to develop at a rapid pace. In recent years, CRISPR-Cas systems other than CRISPR-Cas9 have been discovered and exploited, further completing the CRISPR-Cas genetic toolbox.
The applications of the CRISPR-Cas technology have been extraordinarily wide-ranging, including the generation of transgenic animals, the genetic modification of various eukaryotic cell types, and the genetic modification of plants and crops, among others. Tool and kit service companies offer CRISPR-Cas related products and biotech companies have been founded to develop the technology for the treatment of serious human genetic disorders (e.g. CRISPR Therapeutics). The technology has also attracted the interest of the pharmaceutical and biotech industry, not only to take advantage of its potential to simplify biomanufacturing and screening, but also to apply it to the potential treatment of serious human diseases.
I will take you through the discovery of the CRISPR-Cas9 mechanism and its development as a revolutionary genome engineering technology, discuss the biological roles, mechanisms and evolution of CRISPR-Cas immune systems, as well as more recent aspects and developments of CRISPR-based genome engineering in life sciences and medicine.