HHMI - Precision Genome Editing Enters the Modern Era.
ENPNewswire-June 15, 2020--HHMI - Precision Genome Editing Enters the Modern Era
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Release date- 12062020 - CRISPR has sparked a renaissance in genome editing.
Now, next-generation CRISPR technologies let scientists modify the genome more efficiently and precisely than before.
Such tools could one day serve as therapeutics, but many challenges remain.
During an unusually warm week in January, a hundred scientists from around the world converged in Palm Springs, California, to talk shop. The biologists, geneticists, and chemists came from start-ups and universities, and they had a lot to discuss. Their topic was the genome - or, more precisely, how to alter it.
It was the 1st International Conference on Base Editing, and the mood in the room was electric, says David Liu, a Howard Hughes Medical Institute (HHMI) Investigator at Harvard University. 'The meeting was bustling, people were excited, and it was packed,' he says.
In between talks, scientists ducked outside to eat lunch in the sunshine and share the latest in a field recently kindled and currently blazing. Just three and a half years earlier, Liu and colleagues published a paper describing the first 'base editor,' a tool with the promise of taking the genome editing technology CRISPR to the next level.
With base editors, scientists suddenly had a more efficient and precise way to modify the genome. Such editors could let scientists correct single-letter mutations in DNA - the kind of tiny genetic spelling errors that underlie thousands of human diseases, including sickle cell anemia.
'That was the breakthrough paper,' says Reuben Harris, an HHMI Investigator at the University of Minnesota, who helped organize the January conference. 'It triggered a landslide in this area.' Today, scientists have published more than 300 papers on the technique, used in organisms ranging from bacteria to goats.
Now, the field is inching closer to the clinic. Within the last few months, Harris, Liu, and others have reported the kind of practical refinements needed before base editors are ready for therapeutic use.
Already, scientists have seen success in animals. A week and a half after the conference in Palm Springs, Liu jetted to another meeting, in Banff, Canada, called 'Engineering the Genome.' This time, he reported using base editors in mice to correct the genetic error behind progeria - a rare human condition characterized by rapid aging and early death. It's a long-term study that Liu's team and their collaborators at the National Institutes of Health and Vanderbilt University are still wrapping up. But he calls the results so far 'incredibly exciting.'
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