Researchers from Mass General Brigham and Beth Israel Deaconess Medical Center have developed STITCHR, a novel gene editing tool that inserts therapeutic genes into specific genomic locations without causing unwanted mutations. Unlike traditional systems that require both RNA and DNA, STITCHR is RNA-based, simplifying delivery logistics. By inserting entire genes, it offers a one-step solution to gene therapy, overcoming CRISPR's limitations of correcting individual mutations. The findings were published in Nature.

CRISPR has revolutionized gene editing but has limitations, such as its inability to target all genomic locations or correct all mutations, like those causing cystic fibrosis,” said co-senior author Omar Abudayyeh, PhD. “We sought to insert large gene segments to replace faulty ones and target every mutation in a disease with a single construct.”

STITCHR leverages retrotransposons—genetic elements that can move within the genome—using their copy-and-paste mechanism to edit genes precisely. The team screened thousands of retrotransposons and combined the best candidate with CRISPR’s nickase enzyme to create STITCHR.

We believe STITCHR could become a universal approach for genetic disorders," said lead author Christopher Fell, PhD. The team plans to refine the system and move toward clinical applications.

“Studying cell biology provides inspiration for new tools and therapies for both rare and common diseases,” added co-corresponding author Jonathan Gootenberg, PhD.

Source: https://www.massgeneralbrigham.org/en/about/newsroom/press-releases/new-gene-editing-tool-shows-promise-for-treating-diseases-with-multiple-mutations