Researchers from Mass General Brigham and Beth Israel Deaconess Medical Center created STITCHR, a revolutionary gene editing tool that may introduce therapeutic genes into particular regions while avoiding undesired mutations. The system can be completely RNA-based, which significantly simplifies delivery logistics as compared to previous systems that use both RNA and DNA. By inserting a complete gene, the technique provides a one-step solution that overcomes the limitations of CRISPR gene editing technology, which is designed to correct specific mutations, and represents a hopeful step forward in gene therapy.
Their findings were published in the journal Nature.
CRISPR has revolutionized how we think about gene editing, but it has limitations. CRISPR can’t target every location in the genome, and it can’t fix the thousands of mutations present in diseases like cystic fibrosis,
When we started our lab, one of the big things we wanted to figure out was how to insert large pieces of genes, or even entire genes, to replace faulty ones. This would allow us to target every mutation for a disease with a single gene editing construct.
Omar Abudayyeh
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STITCHR harnesses the power of enzymes from genetic components called retrotransposons, which are found in all eukaryotic cells, including mammals, fungi, and plants. They are commonly referred to as “jumping genes” due to their ability to roam about and insert themselves into the genome. The researchers realized that the copy-and-paste process they employ to transmit data may be used to modify genes at precise sites.
The researchers then used a computer approach to screen thousands of retrotransposons for those that could be reprogrammed, which they tested in the lab. They reduced down to a final option, which was paired with the nickase enzyme from the CRISPR gene editing system to aid in the seamless integration of the genes, resulting in the final STITCHR system.
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We’re really excited about STITCHR and its potential clinical and biotechnological applications,
By replacing or supplementing entire genes, we think STITCHR could become a ‘one-size-fits-all’ approach for patients with a genetic disorder.
Christopher Fell
The researchers intend to continue improving system efficiency and are working on translating STITCHR for clinical applications.
By studying basic biology in our cells, we can find inspiration for new tools. These can expand our cell engineering capabilities and lead to creation of new medicines and therapies for both rare and common diseases.
Jonathan Gootenberg
Source: Mass General Brigham
Journal Reference: Fell, Christopher W., et al. “Reprogramming Site-specific Retrotransposon Activity to New DNA Sites.” Nature, 2025, pp. 1-10, DOI: https://doi.org/10.1038/s41586-025-08877-4.
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