What Is CRISPR & Why Is It Important?
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. It is a component of bacterial immune systems that can cut DNA, and has been repurposed as a gene editing tool. It acts as a precise pair of molecular scissors that can cut a target DNA sequence, directed by a customizable guide.
The system is made up of two key parts: a CRISPR-associated (Cas) nuclease, which binds and cuts DNA, and a guide RNA sequence (gRNA), which directs the Cas nuclease to its target. It was discovered in bacterial immune systems, where it cuts the DNA of invading viruses, called bacteriophage, and disables them. Once the molecular mechanism for its DNA-cleaving ability was discovered, it was quickly developed as a tool for editing genomes.
CRISPR is important because it allows scientists to rewrite the genetic code in almost any organism. It is simpler, cheaper, and more precise than previous gene editing techniques. Moreover, it has a range of real-world applications, including curing genetic disease and creating drought-resistant crops.
The History & Discovery Of CRISPR
Today, CRISPR is well-known as a precise gene editing tool, but it took many years for scientists to figure out what it was and how to harness its potential. Let’s discuss the inventors of this gene editing tool, the scientists who championed this technology, and the history behind these breakthroughs.
Who discovered CRISPR?
CRISPR was discovered by Dr. Jennifer Doudna (UC Berkeley, seen on right in image above) and Dr. Emmanuelle Charpentier (Max Planck Unit for the Science of Pathogens, Berlin, left). Their groundbreaking paper, revealing that the CRISPR-Cas9 bacterial immune system could be repurposed as a gene editing tool, was published in the journal Science in 2012.
It wasn’t until 2020 - well after it had been adopted in labs around the world - that Doudna and Charpentier won the Nobel prize in Chemistry for their discovery, becoming the first all-female team to do so.
Other significant contributors include Feng Zhang at the Broad Institute of MIT and Harvard in Cambridge, Massachusetts, who pioneered the use of CRISPR in eukaryotic cells and discovered novel Cas variants, George Church at Harvard Medical School in Boston, Massachusetts, who was among the first to demonstrate its use in human cells, and biochemist Virginijus Siksnys at Vilnius University in Lithuania, who independently discovered the ability of CRISPR to edit genes in other organisms. For more information on these early pioneers, you can check out our blog about prominent CRISPR scientists.
History of CRISPR
While Doudna and Charpentier were the first to adapt CRISPR-Cas9 as a gene editing tool, the history goes back a little further than their 2012 publication. In 1993, Dr. Fransisco Mojica, a scientist at the University of Alicante in Spain, identified repetitive palindromic segments of DNA interspaced with other fragments of genetic material in bacterial genomes. Dr. Mojico gave these regions the name CRISPR, and proposed that they are a component of the bacterial immune system. In 2007, a team of scientists led by Dr. Philippe Horvath experimentally demonstrated Mojica’s theory.
Since its adaptation by Dr. Doudna and Dr. Charpentier, this versatile gene editing technology has progressed rapidly. It has been adapted for many different purposes, including RNA editing, base and prime editing, live imaging, and diagnostics. It has been used to edit DNA in a variety of organisms, including humans.
In 2019, the first CRISPR clinical trials began, harvesting cells from patients with sickle cell disease (SCD) and editing them in vitro before infusing them back into the body - a method known as cell therapy. After the success of SCD cell therapy trials, a CRISPR treatment was injected directly into human patients for the first time in 2020. This technique is known as gene therapy, and was used to treat hereditary blindness.
Негізгі бет CRISPR/ Cas9: Gene Editing tool
Пікірлер