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Revolutionary Gene Edit Promises 'Disease-Agnostic' Treatment
21 Nov
Summary
- New gene-editing strategy targets many rare diseases at once.
- Researchers engineered a molecule to read past faulty stop signals.
- Human cell and mouse models show promising restored protein function.
Scientists have developed a groundbreaking gene-editing technique that could revolutionize treatment for numerous rare genetic diseases. This innovative approach, dubbed PERT (prime editing-mediated readthrough of premature termination codons), bypasses the need to correct each specific genetic mutation.
Instead, PERT utilizes a specially engineered molecule to help cells overcome premature 'stop' signals in genes, allowing for the production of complete proteins. This strategy has demonstrated success in lab models of diseases such as Batten disease, Tay-Sachs, cystic fibrosis, Niemann-Pick disease type C1, and Hurler syndrome, restoring crucial protein function.
While the potential for treating approximately 30% of genetic diseases caused by these stop signals is immense, experts caution that widespread human application is still years away. Significant hurdles remain, particularly in delivering the gene-editing tools effectively to all necessary cells throughout the body, which is critical for managing these complex conditions.
