Nobel Prize for chemistry honors exquisitely precise gene-editing technique, CRISPR – a gene engineer explains how it works

Researchers have been able to manipulate large chunks of genetic code for almost 50 years. But it is only within past dece that y have been able to do it with exquisite precision – ding, deleting and substituting single units of genetic code just as an editor can manipulate a single letter in a document. This newfound ability is called gene editing, tool is called CRISPR, and it’s being used worldwide to engineer plants and livestock and treat disease in people.

For se reasons 2020 bel Prize in chemistry has been awarded to Emmanuelle Charpentier , director of Max Planck Unit for Science of Pathogens in Germany, and Jennifer Doudna , professor at University of California, Berkeley, for discovering and transforming CRISPR into a gene-editing techlogy. It’s first time two women have shared a bel prize.

I’m a CRISPR engineer , interested in developing vel CRISPR-based gene-editing tools and delivery methods to improve ir precision and function.

In past, my colleagues and I have created a version of CRISPR that can be controlled using light , which allows precise control of where and when gene editing is performed in cells, and can be potentially used in animals and humans. We’ve also created a targeted system that can pack and deliver editing components to desirable cell s – it’s like GPS for cells. Most recently, we engineered a tool that improved speed and precision of CRISPR so it could be used in rapid diagstic kits for COVID-19, HIV, HCV and prostate cancer .

While CRISPR scientists like me have been speculating about a bel Prize for CRISPR, it was exciting to see Charpentier and Doudna win. This will encour young, talented engineers and researchers to enter field of gene editing, which can be leverd for designing new diagstics, treatments and cures for a range of diseases.

CRISPR/Cas systems as gene editors

Many variants of CRISPR/Cas systems have been discovered, engineered and applied to edit genes. re are alrey over 20,000 scientific publications on topic.

CRISPR dates back to 1987, when a Japanese molecular biologist, Yoshizumi Ishi, and colleagues discovered a CRISPR DNA sequence in E. coli. CRISPR sequence was later characterized by a Spanish scientist, Francisco Mojica, and colleagues, who named it CRISPR, which stands for Clustered Regularly Interd Short Palindromic Repeats .

While people and animals have evolved complex immune systems to fight viral attacks, single-cell microorganisms rely on CRISPR to find and destroy a virus’s genetic material to stop it from multiplying.

Charpentier and Doudna figured out how to borrow this innate biological capability from microbes and apply it to genetic engineering of bacteria.

In a landmark paper, published online on June 28, 2012, Charpentier and Doudna showed that CRISPR gene-editing machinery includes two components: a guide molecule that serves as sort of a GPS to find and bind target gene site on DNA of an inving virus, which n teams up with a CRISPR-associated protein (Cas) that serves as a molecular scissor that snips DNA .

Around same time, Virginijus Siksnys , a Lithuanian biochemist at University of Vilnius, me a similar discovery and submitted results for publication that appeared a few months later, in September 2012 . Feng Zhang , a biologist at Bro Institute in Cambridge, Massachusetts, and colleagues showed that CRISPR can be improved and used for editing mammalian cells . He currently owns one of first patents on using CRISPR for gene editing, which is being contested by Doudna’s institution, UC Berkeley.

Once DNA has been cut in right spot, cell will try to repair cut. But repair mechanism is error prone, and oftentimes cells fail to fix cuts perfectly, ultimately disabling gene. Disrupting a gene is particularly useful for studying its function and find out what happens if you stop a gene from working. This technique is also useful for treating cancer and infections, where turning off a gene can potentially stop cancer cells and pathogens from dividing or kill m outright.

During this cutting-repair process, one can fool cells by providing a new piece of DNA. cells will n incorporate this piece of DNA with desirable edits into genetic code. This enables researchers to correct a genetic mutation that causes a genetic disease, or replace a defective gene with a healthy one.

beauty of CRISPR lies in its simplicity. CRISPR can be easily customized to target any gene of interest, wher it is in plants, animals or people. CRISPR applications range from tools for understanding biology, as diagstics and as new kinds of rapeutics to applications in producing better crops, biofuels and transplantable organs .

[ Conversation’s science, health and techlogy editors pick ir favorite stories. Weekly on Wednesdays .]

Why CRISPR deserved a bel Prize

While re is still plenty of room for improvement of se techlogies, scientists have alrey begun testing CRISPR in a number of clinical trials for treating cancer and genetic disorders. CRISPR-based diagstics have been also been approved by U.S. Food and Drug ministration under emergency use authorization for COVID-19 testing .

CRISPR does come with a lot of ethical concerns that warrant caution. For example, in 2018, a Chinese scientist prematurely and unethically used CRISPR for editing human embryos and created CRISPR-edited babies that could pass se genetic alterations to ir offspring for generations to come. Some have used techlogy for or CRISPR-related DIY biohacks that raise more concerns over regulating gene-editing techlogy .

Despite se concerns, CRISPR has huge potential to transform how scientists can detect , treat and even ericate diseases as well as improve agricultural products. Society is alrey seeing benefits of this bel-winning techlogy.

This story has t been edited by www.republicworld.com and is auto-generated from a syndicated feed.