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A new method for safely inserting large chunks of DNA into genomes has now measured up in mice, potentially paving the way for the next generation of gene editing medicines.
The approach, which is described in a Nature paper published March 11, uses circular, single-stranded DNA rather than the typical double-stranded version of the hereditary molecule. Dubbed “integration through nucleus-synthesized template addition of large lengths”—INSTALL for short—the technique was pioneered by a team led by Connor Tou, Ph.D., and Benjamin Kleinstiver, Ph.D., at Mass General Brigham.
With INSTALL proving capable of inserting DNA into the genomes of mice and lab-grown human cells, the team is now working to use it in new medicines for liver diseases, blood disorders and metabolic conditions, Kleinstiver told Fierce Biotech in an interview.
“This is beautiful work that addresses in an elegant way one of the big challenges with controlled DNA insertion into the genome,” Kiran Musunuru, M.D., Ph.D., co-founder of Verve Therapeutics and a scientist at the Children’s Hospital of Philadelphia and the University of Pennsylvania, told Fierce. Musunuru co-led development of the ...