This year’s Nobel Prize in Chemistry for the creation of the “gene scissors”. What is the CRISPR / CAS9 method? [WE EXPLAIN]

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The 2020 Nobel Prize in Chemistry was awarded to two researchers – Emmanuelle Charpentier and Jennifer A. Doudna, creators of the precision ‘gene scissors’. Their method, mysteriously called CRISPR / Cas9, is to allow for the development of, inter alia, new anti-cancer therapies.

  1. “Genetic scissors” was discovered by the French – Emmanuelle Charpentier, and perfected in cooperation with the American – Jennifer A. Doudna
  2. The discovery is based on the mechanism of the purulent streptococcus immune system, which causes, among others, angina
  3. The CRISPR / Cas9 method allows you to switch off selected genes or modify them
  4. Scientists have high hopes for its use in the treatment of genetic diseases and cancer

Emmanuelle Charpentier and Jennifer A. Doudna developed their method of “molecular scissors” in 2012. Only eight years later they were awarded the Nobel Prize.

What is the CRISPR / Cas9 method?

Overall, selected DNA sequences can be modified using the CRISPR / Cas9 “genetic scissors”.

CRISPR – what is it?

The name CRISPR is an abbreviation made up of the first letters of the words: Clustered Regularly Interspaced Short Palindromic Repeats, that is, in Polish, grouped, regularly interrupted, short palindromic repetitions.

This is a common genetic anomaly that always involves an enzyme that breaks down DNA. This is Cas – short for a system associated with CRISPR. The Cas enzyme can cut a strand of DNA anywhere, like sharp scissors. Such a cut is enough to, for example, neutralize the threat posed by viruses.

The method is extremely precise and can be used to modify the DNA of animals, plants and microorganisms.

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The discovery was made by accident, during the research of Emmanuelle Charpentier (now director of the Max Planck Institute for Infectious Biology in Berlin) on the pathogenic bacterium – Streptococcus pyogenes, or purulent streptococcus, causing, among others, angina. It turned out that the bacterium has a mechanism that was part of the ancient ‘immune system’ of bacteria, CRISPR / Cas. It works by disarming viruses by splitting their DNA.

In 2011, Charpentier began working with Jennifer Doudna, an experienced biochemist at the University of California at Berkeley. Together, they recreated the “genetic scissors” in the laboratory and then perfected them.

In their natural form, they recognize the DNA of viruses, but it has turned out that they can be controlled to cut any DNA molecule at a specific point. Where DNA is cut, genetic code changes can be made.

Why is the discovery of the gene scissors so important?

The method has already revolutionized the life sciences. It provides tools thanks to which we can influence individual genes in living cells. We can change the genetic code in a matter of weeks. It is faster, cheaper, more accurate and more efficient than other genome editing methods known to date.

– The CRISPR / Cas9 method not only revolutionized basic science, but also resulted in innovative crops. It will also lead to new breakthrough treatments, says Claes Gustafsson, chairman of the Nobel Committee.

How can we use CRISPR / Cas9 in practice?

Scientists hope that new anti-cancer therapies and possibly treatments for genetically determined diseases will soon be developed with the help of ‘gene scissors’.

Research in this direction is already underway. It is enough for us to deliver CRISPR / Cas-9 to human cells with the gene we want to switch off, and it will find and neutralize this gene. Additionally, this gene can be modified. We supply the cells we want to change with the gene previously synthesized in the laboratory, and the DNA repair mechanisms themselves stick it in place of the incision made by the Cas protein.

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Already in 2015, a mosquito that did not transmit malaria was created using “genetic scissors”, and a year later in China, human embryos were modified, “teaching” their immune system to recognize malignant lung cancer cells. However, due to ethical and safety concerns, editing of the genome of germ cells and embryos is currently illegal in many countries.

The effectiveness of this year’s award-winning method is also tested in cystic fibrosis, hemophilia and sickle cell anemia, as well as in more complex diseases such as heart disease, mental illness and human immunodeficiency virus (HIV) infection.

Thousands of patents have also been filed for other applications. Thanks to it, plant researchers were able to develop mildew, pest and drought-resistant crops.

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