Gene therapy has shown promise in treating many genetic diseases, but some conditions, like muscular dystrophy, involve very large genes that are too big to be delivered using current methods. A new technology called StitchR has solved this problem and could offer a treatment option for these hard-to-treat conditions.
Instead of trying to deliver the whole large gene at once, StitchR splits the gene into two smaller parts. These two parts are delivered separately into cells, where they join together to form the full gene. This allows the body to make the missing or faulty protein that causes the disease. In animal tests, this method successfully restored proteins like Dystrophin (which is missing in Duchenne muscular dystrophy) and Dysferlin (which is missing in limb-girdle muscular dystrophy).
This breakthrough was made possible by ribozymes—naturally occurring RNA molecules—that help the two parts of the gene come together inside the cells. The StitchR method ensures that only the complete, full-length protein is made, which is a big improvement over other methods that may produce incomplete or ineffective proteins.
The team at the University of Rochester, led by Dr. Douglas M. Anderson, has shown that this technology can work with many different genes and diseases. This discovery could make gene therapy a viable treatment for many conditions that have been difficult to treat until now.
Created: Nov 18th, 2024
Citations:
New Discovery Enables Gene Therapy for Muscular Dystrophies, Other Disorders. (2024, November 14). URMC Newsroom. https://www.urmc.rochester.edu/news/story/new-discovery-enables-gene-therapy-for-muscular-dystrophies-other-disorders
Thomas, U. (2024, November 15). Gene Therapy for Muscular Dystrophy Could Use StitchR to Assemble XL Gene from S Segments. GEN - Genetic Engineering and Biotechnology News. https://www.genengnews.com/topics/genome-editing/gene-therapy-for-muscular-dystrophy-could-use-stitchr-to-assemble-xl-gene-from-s-segments/
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