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New Approach Could Make Gene-Editing Treatments Faster and Cheaper
The PERT gene-editing method restores protein production in lab models and could treat 30% of genetic diseases caused by premature stop signals, researchers say.
- On Nov. 21, 2025, David Liu's team published in Nature a study introducing PERT, a prime editing‑mediated readthrough method to treat many rare genetic diseases, HealthDay reported.
- Nonsense mutations create premature 'stop' signals that prevent full proteins, causing faulty proteins behind more than 7,000 rare genetic diseases affecting fewer than 200,000 people in the United States.
- Using prime editing, researchers inserted a suppressor tRNA that restored protein function in human cell models and a mouse model without causing mistakes or toxicity.
- Experts warn delivering gene-editing tools to all cells, especially in brain and lungs, remains a major obstacle, and an Intellia Therapeutics trial ended with a patient's death, requiring years of testing.
- By removing the need to correct each mutation individually, disease‑agnostic approach could help 10% to 15% of patients, including 200,000 with Stargardt disease and 31,000 with phenylketonuria, experts say.
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Total News Sources23
Leaning Left4Leaning Right3Center5Last UpdatedBias Distribution42% Center
Bias Distribution
- 42% of the sources are Center
42% Center
L 33%
C 42%
R 25%
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