2 scientists win Nobel chemistry prize for gene-editing tool

STOCKHOLM — The Nobel Prize in chemistry went to two researchers Wednesday for a gene-editing tool that has revolutionized science by providing a way to alter DNA, the code of life — technology already being used to try to cure a host of diseases and raise better crops and livestock.

Emmanuelle Charpentier of France and Jennifer A. Doudna of the United States won for developing CRISPR-cas9, a very simple technique for cutting a gene at a specific spot, allowing scientists to operate on flaws that are the root cause of many diseases.

“There is enormous power in this genetic tool,” said Claes Gustafsson, chair of the Nobel Committee for Chemistry.

More than 100 clinical trials are underway to study using CRISPR to treat inherited diseases, and “many are very promising,” according to Victor Dzau, president of the National Academy of Medicine.

“My greatest hope is that it’s used for good, to uncover new mysteries in biology and to benefit humankind,” said Doudna, who is affiliated with the University of California, Berkeley, and is paid by the Howard Hughes Medical Institute, which also supports The Associated Press’ Health and Science Department.

The prize-winning work has opened the door to some thorny ethical issues: When editing is done after birth, the alterations are confined to that person. Scientists fear CRISPR will be misused to make “designer babies” by altering eggs, embryos or sperm — changes that can be passed on to future generations.

Much of the world became aware of CRISPR in 2018, when Chinese scientist He Jiankui revealed he had helped make the world’s first gene-edited babies, to try to engineer resistance to infection with the AIDS virus. His work was denounced as unsafe human experimentation, and he has been sentenced to prison in China.

In September, an international panel of experts issued a report saying it is too soon to try such experiments because the science isn’t advanced enough to ensure safety.

“Being able to selectively edit genes means that you are playing God in a way,” said American Chemistry Society President Luis Echegoyen, a chemistry professor at the University of Texas El Paso.

Dr. George Daley, dean of Harvard Medical School, said: “New technology often presents this dichotomy — there is immense potential for human benefit, especially for disease treatment, but also the risk of misapplication.”

However, scientists universally praised the great potential that gene editing has for patients now.

“There’s no aspect of biomedical research that hasn’t been touched by CRISPR,” which has been used to engineer better crops and to try to cure human diseases including sickle cell, HIV infection and inherited forms of blindness, said Dr. Kiran Musunuru, a genetics expert at the University of Pennsylvania who is researching it for heart disease.

Doudna said CRISPR also has the potential to be used to engineer plants to store more carbon or to withstand extremes of climate change, giving researchers a chance to “address urgent problems humanity is facing.”

It’s the fourth time in the 119-year history

Emmanuelle Charpentier, Jennifer Doudna win Nobel Prize in Chemistry for genome editing tool

Oct. 7 (UPI) — A French scientist and an American professor were awarded the Nobel Prize in Chemistry on Wednesday for their work in developing a “genetic scissors” used to fight human diseases.

Emmanuelle Charpentier, a French-born researcher and director of the Max Planck Unit for the Science of Pathogens in Germany, and Jennifer Doudna, a professor at the University of California, Berkeley, were given the chemistry prize, the Royal Swedish Academy of Sciences announced in Stockholm.

The pair worked together to help develop the CRISPR/Cas9 gene editor, which is capable of cutting DNA genomes in precise locations and allowing researchers to add or delete pieces of genetic material or make changes by replacing an existing segment with a customized sequence.

The tool’s development has led to widespread applications for genome editing — and is faster, more accurate, more efficient and less expensive than other existing methods.

The CRISPR/Cas9 system is used to research a wide variety of diseases, including single-gene disorders including cystic fibrosis, hemophilia and sickle cell disease. It also holds some promise for treating and preventing complex diseases like cancer, heart disease, mental illness and HIV/AIDS.

Genome editing has also found a strong presence in agriculture, where it’s used to develop crops resistant to mold, pests and drought.

After publishing an initial discovery in 2011, Charpentier teamed with Doudna to develop the “genetic scissors” to make it easier to use. The next year, they proved it could be controlled and used to cut any DNA molecule at a predetermined site.

“There is enormous power in this genetic tool, which affects us all,” Claes Gustafsson, chairman of the Nobel Committee for Chemistry, said in a statement Wednesday. “It has not only revolutionized basic science, but also resulted in innovative crops and will lead to ground-breaking new medical treatments.”

Due to ethical considerations, scientists are limited to using CRISPR/Cas9 in humans on somatic cells — cells other than egg and sperm cells. Changes made in those cells are not passed from one generation to the next.

The Nobel Prize for medicine was awarded Monday to Americans Harvey J. Alter and Charles M. Rice and Briton Michael Houghton for their work on curing Hepatitis C; the prize for physics was given Tuesday to Roger Penrose, Reinhard Genzel and Andrea Ghez for their research on black holes.

The Nobel Prize in Literature will be awarded Thursday, the peace prize on Friday and the prize for economic sciences on Oct. 12.

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