French microbiologist Emmanuelle Charpentier, US molecular biologist Jennifer Doudna and US biological engineer Zhang Feng (張鋒) yesterday won the Tang Prize in biopharmaceutical science for the development of a breakthrough genome editing platform with the potential to revolutionize disease treatment.
The three contributed to the development of a genome editing system known as CRISPR/Cas9 — clustered regularly interspaced short palindromic repeats/CRISPR associated protein 9 — and they are to share a cash prize of NT$40 million (US$1.24 million) and a research grant of NT$10 million, Nobel laureate Lee Yuan-tseh (李遠哲), who chairs the Tang Prize Selection Committee, told a news conference in Taipei.
First discovered in bacteria in the 1980s, the CRISPR system was later identified as a bacterial defense mechanism against viruses and foreign DNA.
The system helps the bacterial genome “memorize” viral DNA to recognize and defend against bacteriophages, while nucleases encoded with CRISPR-associated genes would be responsible for “cutting” the virus.
The three scientists have applied the system to genome engineering to cut any desired gene.
Charpentier first identified two strands of ribonucleic acid (RNA) that could guide the Cas9 nuclease to target genes, and later worked with Doudna to fuse those two strands into a single programmable guide RNA to be delivered into a cell, allowing the cell’s genome to be cut at any desired location, Academia Sinica research fellow Kung Hsing-jien (龔行健) said.
“If we have to single out just one of Charpentier’s many contributions, it is the original discovery of the ‘GPS system’ for Cas9, which can be used to cut existing genes or add new genes,” he added.
Meanwhile, Zhang, who was working independently, reported the first successful implementation of Cas9-based genome editing in mammal and human cells.
The system’s potential medical applications include repairing defective genes that cause hereditary diseases and fixing mutated genes that cause cancer development, Kung said.
“It has always been the dream of scientists to someday effectively edit the human genome for medical purposes, and because of the brilliant work by the recipients of this year’s Tang Prize, that someday is today,” Kung said.
Hundreds of research facilities worldwide have implemented the CRISPR/Cas9 system in experiments with different animal and plant species, Kung said, adding that some regard it to be one of the greatest advances in genomics since the development of cloning and polymerase chain reaction.
“There are several genome editing techniques, but CRISPR/Cas9 is much more precise and efficient than the others,” Kung said.
“It used to take more than a year to develop genetic edits for mice, but CRISPR reduces development time to a few weeks with an 80 percent success rate,” he added.
“Defective genes that play a critical part in cancer development are usually found in a tiny ‘hot spot.’ Although it is possible to remove the hot spot, doctors and scientists lacked the necessary tools to do so,” Taipei Medical University president Yen Yun (閻雲) said.
“What these three scientists have given us is a pair of scissors, a spotlight and a GPS system to significantly improve [future cancer treatments],” Yen added.
Lee said the system could also be used to edit out corn genes responsible for growing starch that cannot be digested by humans.
The Tang Prize was established in 2012 by entrepreneur Samuel Yin (尹衍樑) to honor the top researchers and leaders in sustainable development, biopharmaceutical science, sinology and rule of law. The first prizes were awarded in 2014. The winners of this year’s sinology prize and law prize are to be announced today and tomorrow respectively.
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