Investigators from Mass General Brigham and Beth Israel Deaconess Medical Center have developed STITCHR, a new gene editing tool that can insert therapeutic genes into specific locations without causing unwanted mutations.
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American researchers have unveiled STITCHR in Nature, a novel gene-editing tool that enables the precise insertion of entire therapeutic genes into specific genomic locations without introducing unwanted mutations. Unlike traditional CRISPR systems, which typically correct single mutations and often face targeting limitations, STITCHR offers a "one-and-done" solution by inserting full genes. It may be a game-changing approach for treating diseases caused by a wide range of mutations, such as cystic fibrosis. STITCHR, short for Specific Target-Primed Insertion Through Targeted CRISPR Homing of Retroelements, harnesses enzymes derived from retrotransposons, genetic elements also known as "jumping genes", to guide gene integration with high specificity. Notably, the system can be delivered entirely in RNA form, simplifying logistics compared to approaches that require both RNA and DNA components. This advancement marks a major step forward in gene therapy, offering a promising strategy to treat diverse genetic disorders by replacing faulty genes in their entirety.