Tech Tuesday: What is CRISPR?
With increasing amounts of research into various diseases and disorders stemming from genetic mutations, scientists have begun to investigate ways to directly change the genetic makeup of cells.
Genetic disorders occur in about 2 to 3 percent of newborns, 5 percent of the adult population below the age of 25 and 60 percent in later life, according to the Genetic Alliance UK website.
The lives of individuals afflicted with genetic disorders such as muscular dystrophy can be very difficult, developing problems with mobility, diet and sleep, according to the Parent Project Muscular Dystrophy website.
These disorders arise from the DNA, where any kind of mutation or defective gene can lead to immense issues for the person it is in. Genetic mutations can be passed on for generations, leading to the spread of the disorder, according to the National Human Genome Research Institute website.
Disorders such as sickle-cell anemia, cystic fibrosis, hemophilia and muscular dystrophy are all genetic disorders, according to the website.
Rather than research drugs to treat these disorders, some scientists are working to directly change the genetic makeup of a cell to one day remove the consequences of genetic disorders, according to the Nature Publishing Group website, a scientific publication.
The method gaining immense attention over the last few years is Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, which can edit gene function and expression, according to the website for Addgene, a non-profit organization that helps scientists share genetic information.
Genes are the information carried by DNA. DNA is a double stranded helix, with a single stranded counterpart called RNA. Both DNA and RNA are made through combinations of 5 nucleotides, according to the Boise State University website.
A long process must take place to express a gene. First, the DNA corresponding to the desired gene is transcribed into RNA, which is processed so areas that do not code for everything is removed. That product is transported outside the cell nucleus and translated into proteins, according to the Nobel Prize website.
Before CRISPR, scientists had to create a new nuclease-pair for every target on the genome. The nuclease-pair would be directed to the desired spot on the gene, but was created in a meticulous process, according to the Addgene site.
CRISPR is made of two parts, a “guide RNA” (gRNA) and an endonuclease related to CRISPR (Cas9). The gRNA is a short RNA strand with a sequence of nucleotides that allows Cas9 to to bind to it, as well as a sequence that describes the target gene, according to the site.
Changing the target gene sequence allows the scientist to find and influence any gene they like, and gives great power to researching new things, according to the site.
CRISPR was initially designed to strictly knockout, or remove from activity, specific genes. Over time, modifications have made it possible to activate or repress the activity of genes and even take images of live cell DNA using fluorescence, according to the site.
Despite CRISPR’s massive potential, it has sparked debate regarding the ethics and safety of some of the experiments it is used in, according to the Nature Publishing Group website.
Some issues include whether or not it should be used to make changes to the human genome and what some of the potential ecological consequences of genomically editing organisms, according to the site.
Nonetheless, CRISPR has had an explosive increase in research usage over the last three years, and it is only expected to get higher, according to the site. Especially considering the significantly cheaper price and simple methods of CRISPR, research should be expected to expand and gain more attention in the future.
Harshel Patel is a School of Arts and Sciences sophomore majoring in molecular biology and biochemistry. He is the digital editor at The Daily Targum. He can be found on Twitter @harshel_p.