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Scientists have achieved a historic medical breakthrough by using gene editing directly inside a living newborn to treat a rare and deadly genetic disorder. The baby, diagnosed with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, couldn’t break down ammonia, which threatened his brain and liver. Traditional treatments like liver transplants weren’t an option because of his age. So, researchers across the U.S. developed a custom CRISPR gene therapy tailored to fix the baby’s specific g
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Scientists have achieved a historic medical breakthrough by using gene editing directly inside a living newborn to treat a rare and deadly genetic disorder. The baby, diagnosed with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, couldn’t break down ammonia, which threatened his brain and liver. Traditional treatments like liver transplants weren’t an option because of his age. So, researchers across the U.S. developed a custom CRISPR gene therapy tailored to fix the baby’s specific g
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