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Harvard, MIT Researchers Develop New Gene Editing Technology

Scientists at the Broad Institute in Kendall Square have made advances in gene-editing technology in recent years.
Scientists at the Broad Institute in Kendall Square have made advances in gene-editing technology in recent years. By Owen A. Berger
By Virginia L. Ma and Meera S. Nair, Contributing Writers

A team of researchers from the Broad Institute led by professor of Chemistry and Chemical Biology David R. Liu ’94 developed a novel gene-editing technique called “prime editing.”

The technique, published in the scientific journal Nature on Monday, could “correct about 89% of known pathogenic human genetic variants,” with broad applications in the field of medicine, according to Nature.

Liu and his team’s research has captured the attention of scientists and experts in the field.

Harvard Medical School genetics Professor George M. Church wrote in an email that the research from Liu’s lab has made significant progress from previous gene-editing technologies.

“This prime-editing method is much, much closer to the grail of unlimited and precise edits than any simple nuclease,” Church wrote.

The technique could have larger implications on the field of gene editing, according to Fyodor Urnov, a biology professor at the University of California, Berkeley and scientific director at the Innovative Genomics Institute.

“I think it could become a tool of major impact in the arsenal that gene editors have to engineer the cell and organism,” Urnov said.

Still, the field of gene editing continues to face ethical questions. The technology could be used to enhance human performance, according to Urnov.

Prime editing implements a search-and-replace tactic that “is capable of directly editing human cells in a precise, efficient, and highly versatile fashion,” according to a Broad Institute press release. This allows scientists to create all possible types of point mutations, insertions, and deletions, whereas prior iterations of gene-editing technologies did not have this capability.

“If CRISPR-Cas9 and other programmable nucleases are like scissors, and base editors are like pencils, then prime editors are like word processors, capable of searching for target DNA sequences and precisely replacing them with edited DNA sequences,” Liu wrote in an email.

Currently, there are few viable treatments for rare genetic diseases like cystic fibrosis and sickle cell disease. Prime editing could potentially contribute to treatments for these diseases, according to Urnov.

Andrew V. Anzalone, a postdoctoral fellow and first author on the paper, said the lab has used their new technique to edit mammalian cells in a petri dish.

This capability has applications in the field of therapeutics, as researchers can study effects of “changes to genes” or model “biological pathways,” according to Anzalone.

Liu wrote that the ultimate goal of gene editing is “to be able to make any DNA change, in any position of a living cell or organism, including human patients with genetic diseases.”

“Much more work is needed to fully realize this goal,” he wrote.

The development of this technique comes at a time when the Broad Institute is involved in a separate dispute over CRISPR Cas9 patent rights with UC Berkeley, which alleges that the Broad used “deceit” to win patent rights to CRISPR-Cas9 in eukaryotic cells

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