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Gene Editing

CRISPR genome editing gets 2020 Nobel Prize in Chemistry

Emmanuelle Charpentier and Jennifer A. Doudna share award

by Ryan Cross
October 9, 2020 | APPEARED IN VOLUME 98, ISSUE 39


Credit: Vilnius University (Charpentier); Laura Morton Photography (Doudna)
Emmanuelle Charpentier (left) and Jennifer A. Doudna

The 2020 Nobel Prize in Chemistry has gone to Emmanuelle Charpentier and Jennifer A. Doudna “for the development of a method for genome editing.” That method, formally known as CRISPR-Cas9 gene editing but often called simply CRISPR, allows scientists to precisely cut any strand of DNA they wish. In the 8 years since its creation, CRISPR has been a boon for biologists, who have published thousands of studies showing that the tool can alter DNA in organisms across the tree of life, including butterflies, mushrooms, tomatoes, and even humans.

“The number of discoveries in biomedicine that have had the impact that Jennifer’s and Emmanuelle’s had can be counted on the fingers of one hand: recombinant DNA, PCR [polymerase chain reaction], DNA sequencing, and now CRISPR,” says Fyodor Urnov, a gene-editing scientist at the University of California, Berkeley. “We have never had a technology as powerful and versatile as genome editing with CRISPR.”

Charpentier, who is now at the Max Planck Unit for the Science of Pathogens, and Doudna, at UC Berkeley, began working together in 2011. The two scientists were inspired by a little-studied bacterial immune system that uses an enzyme called Cas9 to chop up the genes of invading viruses, which are saved as molecular mug shots. In 2011, Charpentier worked out the details of how a pair of bacterial RNA molecules controlled this process (Nature 2011, DOI: 10.1038/nature09886).

Credit: Adapted from OriGene Technologies
CRISPR gene editing uses a synthetic guide RNA, which is complementary to a target DNA sequence, to direct Cas9 to a specific location for cutting. Some applications require an additional DNA template (not shown) to fill in the cut.

The two scientists began thinking about how they could rework this viral defense system into an easily programmable gene-editing tool. They synthesized a new molecule, called the single-guide RNA, which combines key features of the two bacterial RNAs and directs Cas9 to cut a specific site in DNA (Science 2012, DOI: 10.1126/science.1225829).

It was the tool that scientists had been waiting for. CRISPR is cheaper, faster, and easier to use than previous gene-editing tools. Academic labs quickly adopted the technique. Today, scientists can order Cas9 and custom guide RNAs at the click of a button. CRISPR opened up gene editing to the masses.

Charpentier, Doudna, and many others have founded biotech companies to apply CRISPR to agriculture and medicine. A few firms have already begun testing experimental therapies that use CRISPR to treat, and potentially cure, rare genetic diseases and cancer in humans.

“It’s wonderful to see Jennifer and Emmanuelle recognized in this way,” says David Liu, a CRISPR scientist at Broad Institute of MIT and Harvard. “With new treatments for human genetic diseases already in patients, with early positive outcomes, the era of human genome editing has already begun, and Emmanuelle and Jennifer are two of the key pioneers responsible for initiating this new era."



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Eleftherios Pavlides (October 10, 2020 12:36 AM)
What a heartwarming story with immense possibilities for application in so many areas!

One that may prove of the utmost importance is in energy. CRISPR may put the the last death nail to the coffin of the dying dirty fossil fuel economy. Ironically more lives may be saved not by using CRISPR therapeutically but preventively, by eliminating disease causing pollution. Helping to usher the clean renewable inexpensive hydrogen will bring about the third leg of the trifecta wind - solar - hydrogen to put an end to the threat of global climate holocaust.

There are several technologies out there closing in on creating enough inexpensive hydrogen to eliminate the need for even a drop of petroleum, a cubic millimeter of fossil gas, or even cinder of coal. One of the most promising dark horses is an international effort to improve the efficiency of photosynthesis by up to 300% for increased photosynthetic productivity and hydrogen production from algae. It is led by Anastasios Melis, an American biologist at the University of California, Berkeley who elucidated the possibility of creating hydrogen from algae. By starving it it makes it fall into an earlier anaerobic state that makes algae start emitting hydrogen instead of oxygen.

CRISPR technology may unleash the power of biology to create clean renewable energy that may earn Emmanuelle Charpentier and Jennifer A. Doudna a second Nobel. This time a Peace Nobel.
Esme (October 11, 2020 12:10 PM)
Congrats to these scientists for creating a new unethical, irresponsible GMO technology that is now in the hands of BigAg/Pharma poisoning people and the planet for profit.

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