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Three Noteworthy Applications of CRISPR

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Only 10 years ago, Nobel prize-winning work introduced CRISPR, or clustered regularly interspaced short palindromic repeats, as a new and powerful genome editing tool. Since then, many exciting applications of the CRISPR-Cas9 system have been developed and proposed, giving a taste of what is yet to come.


Progress has occurred at an unprecedented pace. The amazing breadth of applications supports the claim that the researchers made at the time of the original publication: that CRISPR would have a revolutionary impact on the way we are able to utilize DNA science. These applications have the potential to drastically influence the treatment of health conditions, the drug discovery process and even global climate change efforts. Here are three noteworthy applications: 


Preventing heart attacks 


Because heart attacks are one of the leading causes of death across all populations, finding innovative methods for preventative and reactive measures is imperative. Researchers at Verve Therapeutics Inc. have partnered with biotech companies to use genome sequencing to examine heart attack health outcomes in patients with familial hypercholesterolemia. Elevated cholesterol is a significant risk factor for cardiac disease and individuals with familial hypercholesterolemia are at particular risk. Using CRISPR, researchers are targeting two different genes to mitigate cardiac risk—PCSK9 and ANGPTL3. These genes are directly involved in the production of cholesterol in the body, which Verve researchers aim to turn off. Interestingly, this only takes altering or removing one letter of the genome.


Given that this is one of the first trials to explore using CRISPR and DNA editing directly in a human, long-term effects are still a big unknown—which is important since, when genome editing is used in medical applications, changes made to the DNA are permanent. Integrated DNA Technologies’ rhAmpSeq CRISPR Analysis System, for Research Use Only, is one example of a technology that is contributing to the knowledge of unwanted off-target effects of this form of genetic editing. A recent study found that this tool successfully helped annotate indels and homology-directed repair events associated with CRISPR.   


Involving CRISPR in the drug discovery process is expected to accelerate the speed that new treatments for life-threatening conditions can be developed. Promising studies continue to show how this concept is becoming a reality and how it holds the potential for rewriting the future of medicine as we know it.


Influencing disease progression


Disease progression is one area where genetic editing has been proven to be helpful. An example is how it has been used with Japanese encephalitis, a brain infection mostly found in southeast Asian countries, that is caused by a virus transmitted via a bite from an infected mosquito. It is not transmissible from person to person and its prevalence has increased in recent years.


With no viable treatment options, researchers have turned to CRISPR to find a solution for reducing or eliminating the presence of Japanese encephalitis in the general public. Studies as recent as 2020, published in BMC Genomics, describe how CRISPR can assist with genetic profiling for virus-induced neuroinflammatory responses. Specifically, another study from Cell Host & Microbe suggested that Cas12-based (targeting DNA) and Cas-13-based (targeting RNA) methods could be used for both novel diagnostics and even therapeutics for this class of viral disease. Using these methods to target mosquitos that carry the virus for genetic editing, scientists will be able to create more effective vaccinations and other preventative treatments.


Even though it is challenging to predict the direction of a virus as it evolves, CRISPR offers a potentially feasible option for influencing the evolution of Japanese encephalitis and other similar infections.


Combating climate change


As climate change continues to be a major concern for the world, scientists are on a quest to find alternative ways to create a more efficient backbone for food production. Genetically editing plants using CRISPR is a well-validated procedure that scientists have used for years (primarily using Cas9 and Cas12a). Genome editing can also be used to modify genes to improve heat and drought tolerance in plants.


Cas12b is a newer CRISPR system that can target multiple genes in one step during the genetic editing process. Because it allows researchers to address more than one DNA sequence at a time, more nuanced trials can be conducted to examine gene expression. The University of Maryland College of Agriculture & Natural Resources found that the Cas12b system is relevant to transcriptional activation, multiplexing during genomic editing, and transcription interference binding. Results from their projects contribute to the fact that CRISPR is a useful tool for both altering genetic traits and repressing or activating isolated genes, giving scientists the chance to use what DNA is already giving them.


In agriculture, both old and new CRISPR methods serve as a way for researchers to investigate different diseases and invasive pests. Currently, efforts to increase abiotic stress tolerance, manage harmful diseases, improve crop yield and enhance nutrition are part of the focus to create the potential for genetically modified plants that can withstand climate change or other adverse natural events.


A look toward the future of CRISPR


Looking at these examples of what CRISPR can help researchers accomplish, it is clear that there is no end to the possibilities of what gene editing technologies can offer. Although much has already been achieved in the first decade since the discovery of CRISPR as a genome editing tool, technology will continue to evolve and expand on what we already know. In the next decade, life science researchers can expect more innovations with CRISPR and other genetic editing tools. A priority moving forward will be to continue to find ways that ensure these tools can be leveraged safely and effectively. As the technology behind genome editing evolves, more applications will undoubtedly be uncovered—leading to more discoveries that can positively change our world.


About the author: 

Demaris Mills is president of Integrated DNA Technologies, a global genomics solutions provider driving advances that inspire scientists to dream big and pursue breakthroughs. Mills has nearly 20 years of broad global experience in the life sciences, genomics and diagnostics industries, and a proven track record of driving high-impact results while fostering an inclusive, collaborative and highly engaged team environment.


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