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CRISPR is used in landmark treatment to correct genetic misspelling of a single patient
In a world first, scientists used CRISPR to fix a baby's unique genetic mutation. The report offers hope for personalized treatments of rare diseases.
For the first time, scientists say they have reached into the genome of a severely ill child and rewritten the unique misspelling in his DNA.
The results, published in the New England Journal of Medicine on Thursday, are a landmark in the 50-year quest to read and repair the code of life. The boy, a now 9.5-month-old named KJ, was diagnosed days after birth with an ultra-rare disease that impairs his liver’s ability to process ammonia, which can build up and cause permanent brain damage or death.
