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Getting the latest healthcare news for you
Getting the latest healthcare news for you
Stanford scientists have developed TIGRa, an ultracompact gene activation tool that's less than half the size of comparable CRISPR-based tools — and potentially more powerful. Small enough to fit inside viral delivery vectors with room to spare, TIGRa successfully preserved vision in mice with retinal injuries. Researchers say it could eventually be applied to a wide range of diseases, from neurodegeneration to cancer.
Stanford Medicine scientists have unveiled TIGRa ("tiger A"), a miniaturized gene activation tool that could overcome one of gene therapy's biggest hurdles: size. Standard tools like CRISPR are too bulky to be efficiently packaged into the viral vectors used to deliver therapies into cells. TIGRa, based on a newly discovered gene-targeting system called TIGR-Tas, is less than half the size of comparable CRISPR activators — yet in lab tests, it matched or outperformed CRISPR in activating six out of nine therapeutic genes tested.
In a mouse model of retinal injury, TIGRa was injected into the eyes and programmed to activate two protective genes in retinal ganglion cells — the nerve cells damaged in glaucoma and other degenerative eye diseases. Treated mice retained about a third of their vision after injury, with effects lasting four months. The researchers also demonstrated TIGRa's ability to activate up to 12 genes simultaneously, including reprogramming adult cells into stem cells — a task requiring seven genes at once.
Key Takeaways:
Why it matters: Gene activation therapies have long been constrained by the sheer size of molecular tools like CRISPR. TIGRa's compact design and high efficiency could unlock new treatment possibilities for conditions ranging from retinal degeneration and muscular dystrophy to heart disease and cancer.
