In recent years, CRISPR has gained a reputation as one of the most effective approaches to gene editing.
Here are eight things to know about CRISPR and its potential influence on disease prevention.
1. CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, is a gene-editing technology that enables scientists to edit an organism's — such as a human's — DNA. The CRISPR system allows scientists to add, remove and replace segments of genetic material within a living organism's genome, according to the National Institutes of Health's Genetic Home Reference guide.
2. CRISPR is based on the gene-editing systems bacteria use to defend against viruses. Bacteria create DNA segments known as "CRISPR arrays" based on "snippets" of DNA from a virus, according to the NIH guide. The bacteria use these DNA segments to produce RNA that targets the virus. In a laboratory setting, researchers are likewise able to create RNA that targets a specific DNA sequence and enzyme within the genome.
3. Many scientists consider the CRISPR-Cas9 system — which creates modified RNA segments that bind to the CRISPR-associated protein 9 enzyme — to be one of the most precise and least expensive gene-editing techniques. Unlike other approaches, the CRISPR-Cas9 system is able to cut DNA segments without additional tools and can target multiple genes at the same time, according to a guide created by Cambridge, Mass.-based Broad Institute of MIT and Harvard.
4. In a study published in Nature Biotechnology Feb. 6, a team of researchers used gene-editing tech to enable mice suffering from hereditary deafness to hear. In a separate study published in Nature Aug. 2, researchers used CRISPR-Cas9 to "correct" a gene mutation in human embryos that causes hypertrophic cardiomyopathy, a genetic heart disease that sometimes leads to sudden cardiac death and heart failure.
5. Some researchers think CRISPR might hold the key to disease prevention for a variety of medical conditions. In a 2015 study published in Nature Biotechnology, researchers explained a targeted technique they engineered to disrupt genes related to select mosquitos' fertility in an effort to one day "suppress mosquito populations to levels that do not support malaria transmission."
6. In 2015, Science named CRISPR its "breakthrough of the year." "The biomedical applications of CRISPR are just starting to emerge," the magazine published at the time. "In short, it's only slightly hyperbolic to say that if scientists can dream of a genetic manipulation, CRISPR can now make it happen." The technology has continued to gain popularity as additional research findings about its uses are published.
7. However, despite CRISPR boasting one of the most precise approaches, gene-editing technology also comes with its risks. In a study published in Nature Methods June 14, a research team noted they witnessed "unexpected mutations" in secondary genes while attempting to use CRISPR-Cas9 to restore sight in blind mice.
8. As CRISPR's advancements have begun to unfold, ethical concerns over the misuse of gene editing have also grown.
"Scientists have long feared the unforeseen medical consequences of making inherited changes to human DNA," according to The New York Times. "Some experts have warned that unregulated genetic engineering may lead to a new form of eugenics, in which people with means pay to have children with enhanced traits even as those with disabilities are devalued."