CRISPR, the latest genome-editing technology, has been making the headlines in almost every major science journal, heralding a “new era” for science. In a recent article in Nature, CRISPR was referred to as “the biggest game changer to hit biology since PCR.” Why is CRISPR such a big deal? Better yet, what is CRISPR?
CRISPR, which stands for clustered regularly interspaced short palindromic repeats, is actually a naturally-occurring defense mechanism found in bacteria to protect themselves from viruses. It turns out CRISPR is part of the bacteria’s immune system, which keeps small pieces of dangerous viruses around so it can detect and defend against those viruses the next time they attack—much like our own immune system (think of vaccines). The second part of the system is involves a set of enzymes called Cas, which stands for CRISPR-associated proteins. Together, Cas is an enzyme that cuts the DNA and CRISPR is a collection of DNA sequences that tells Cas exactly where to snip. Okay, now that you know what CRISPR is, you’re probably thinking, “how will it affect me?”
CRISPR has been making waves in biotechnology and the health sector. This past October in 2015, Harvard researchers used CRISPR to simultaneously alter 62 genes in pig embryos, creating animals that could, at least in theory, grow human organs for transplant. One biotechnology start-up, Editas, has plans to use CRISPR to correct DNA disorders that affect children and adults. Editas stated this past November in 2015 that they will try to treat one form of a rare eye disease called Leber congenital amaurosis, which affects the light-receiving cells of the retina. Moving through the timeline of CRISPR, this past December in 2015, researchers used CRISPR to treat an adult mouse model of Duchenne muscular dystrophy. This marked the first time that CRISPR successfully treated a genetic disease inside a fully developed living mammal with a strategy that has the potential to be translated to human therapy. But what if I told you CRISPR has already made history in humans? Just two months ago in March, scientists at Temple University edited HIV-1 DNA out of the genome of human immune cells, preventing virus replication and reinfection of the cleared cells.
But perhaps the most impressive use for CRISPR has come this past month, when researchers made a significant improvement—a new CRISPR system that can switch single letters of the genome cleanly and efficiently, in a way that they say could reliably repair many disease-causing mutations. We all know that the genome is a combination of A, C, T and G, so there are 12 possible changes CRISPR can make (A to C, A to T, A to G, etc.). This new system can make just 2 right now: C to T and G to A–however, “at least 3,000 inherited diseases are the result of a C that should be a T or a G that should be an A, including Fanconi anemia and some cancers,” said Harvard biochemist David Liu, who led the work.
It’s a bit close-minded to think that the effects of CRISPR will be felt only in health and medicine. CRISPR’s reach will almost certainly be much greater–mainly because many industries now rely on genetic engineering. Scientists have already begun to use CRISPR to make alternative sources of fuel. In agriculture, companies are using CRISPR to make crops more pest and drought resistant in place of harmful pesticides. Farmers and livestock breeders can utilize CRISPR to produce animals with increased muscle mass and leaner meat. Large food companies, such as Dannon, have begun to use CRISPR to create strains of bacteria that produce more flavorful yogurt and other fermented foods. The possibilities for CRISPR are truly endless, which should make everyone excited to see what can come next–the future is here! At NAG it is a collaborative effort; we are excited to see where this new age of science can take us, and where our technology can help advance science and personalized medicine.
Nevertheless, in order to understand which mutations a person has, they need to have their genomes sequenced. New Amsterdam Genomics is striving to make medicine personalized and intimate. If CRISPR is able to repair disease-causing mutations, it’s imperative that people understand their genome first by getting it sequenced and analyzed. At New Amsterdam Genomics, we are leading the genomics revolution by truly trying to understand our patients, and with CRISPR on the rise, doctors might finally be able to take curative action when it comes to diseases.