CRISPR Cas9 is a revolutionary genome editing technology identified by scientists in past decades. It was invented in 2012 by scientists at the University of California, Berkeley and still, studies are being done to discover more advantages of this technology. CRISPR Cas9 is a unique technology that enables efficient and precise genomic modifications in a wide variety of organisms and tissues.
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeat) are sections of genetic code containing short repetitions of base sequences followed by spacer DNA segments. Cas9 (CRISPR Associated protein 9) is an endonuclease which acts as a molecular scissor to introduce a double strand break at a particular location within a strand of DNA using a guided RNA. CRISPR Cas9 system can be used to edit parts of the genome by removing, adding or altering sections of the DNA sequence. Currently, CRISPR Cas9 is the simplest and most versatile method of genetic manipulation when compared to previous techniques of editing DNA. Other techniques such as zinc finger nucleases and TALENS both require scientists to create custom proteins specific for each DNA target which requires much more effort than the RNA programming needed for CRISPR.
This system is a microbial adaptive immune system found in some bacteria enabling the organisms to respond to and eliminate invading genetic material. Scientist adapted this system so that it could be used in other cells. Researchers have reported success using CRISPR Cas9 in animal embryos, including those of monkeys, mice, fish, and rabbits as well as human stem cells and immune cells. Simply put, targeting efficiency of Cas9 and its ability for modification and adaptation makes this system amenable for genome editing.
Application of CRISPR Cas9 in Health and Medicine
This building success can open up the path to a healthy world with its incredible implications on health sector by preventing diseases, extending quality of life and life expectancy. CRISPR Cas9 has a lot of potential as a tool which can be applied for both drug discovery and therapeutic treatments. It can be used to screen for the activity of genes or functional domains of proteins. Researchers are already using it to identify the targets in oncology by taking a cancer cell and screening every gene in the genome.
CRISPR Cas9 allows targeting the underlying cause of a disease and possibly treating it by modification of the patient’s genome. Soon CRISPR is likely to be developed and approved for the treatment of genetic disorders like Beta Thalassemia and Huntingdon’s disease more effectively. Medicine has not yet adapted to genetic revolution because scientists were unable to analyze genomic data and act according to it. CRISPR Cas9 will bring the solution to this problem because it is the first viable tool which enables us to explore the genome and create treatments to repair the genome in diseases where limited treatment options are available.
Today scientists and researchers are putting their maximum efforts to bring a rapid progress in developing CRISPR Cas9 system into a remarkable technology which can bring about positive developments in medicine and genomic editing. Thus, in the near future, we may have the chance to extend our quality of life with this new technology.
Addgene: CRISPR/Cas9 Guide. (2017). Addgene.org. Retrieved from https://www.addgene.org/crispr/guide/
Hsu, P. D., Lander, E. S., & Zhang, F. (2014). Development and Applications of CRISPR-Cas9 for Genome Engineering. Cell, 157(6), 1262–1278 from https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4343198/