CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology is a simple but powerful tool for genome editing. This tool enables life science researchers to easily edit DNA sequences and modify gene function. It has many potential applications include correcting genetic defects, treating and preventing the spread of diseases and improving crops. By delivering the CRISPR enzyme Cas9 nuclease coupled with synthetic guide RNA (gRNA) into a cell, the cell's genome can be cut at a desired location, that allows existing genes to be removed or add new ones.
What can CRISPR do?
In theory, CRISPR could be applied to modify the DNA of virtually any living being for all sorts of different applications. In biotech and pharma companies, CRISPR is becoming the go-to tool for drug discovery. In academic research labs, the gene editing tool has already become quite popular and is being used by many to modify the genome of organisms ranging from bacteria and worms to mice, pigs, and even monkeys. This is very valuable to understand the function of any gene of interest, whether it is one that causes disease or one that makes a crop produce better yields or survive harsh conditions.
In agriculture, CRISPR could be used to produce crops that grow faster or that resist drought much faster than with traditional breeding techniques. It can also be used to add new features, such as making tomatoes spicy, or remove others — for example making gluten-free wheat or decaf coffee beans. However, regulations can limit the use of these technologies. While the US has already seen the launch of CRISPR-modified crops, the European Union decided to set strict GMO regulations that scientists believe are hindering the potential of the technology.
But right now, most of the money seems to be in using CRISPR-Cas9 to engineer human DNA. With over 10,000 diseases caused by mutations in a single human gene, CRISPR offers hope to cure all of them by repairing whatever genetic error the patient has.
There are two main approaches to using CRISPR as a human therapeutic. The first is called ex vivo gene editing, and involves extracting human cells, engineering them in the lab, and reinjecting them into the patient. This method is similar to that used for most gene therapies already in the market, and it allows more control over the process. However, it can become quite expensive given each patient requires an individual manufacturing process for their therapy.
The second method is called in vivo gene editing and involves delivering CRISPR-Cas9 into the patient’s body to edit the DNA directly from within the cells. CRISPR could be delivered inside nanoparticles or encoded into DNA and be cleared out of the body once it has completed its mission.
Companies Working In CRISPR Technology
Thermo Fisher Scientific Inc., Merck KGaA, Horizon Discovery Group plc, Cellecta, Inc, GeneCopoeia, Inc., New England Biolabs, OriGene Technologies, Inc., GenScript, Integrated DNA Technologies, Inc. and Agilent Technologies, Inc. And Others