A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the rare condition carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after birth, has responded positively to the treatment. The process, from diagnosis to treatment, took only six months and marks the first time the technology has been successfully deployed to treat a human patient. The technology used in this study was developed using a platform that could be tweaked to treat a wide range of genetic disorders and opens the possibility of creating personalized treatments in other parts of the body.
Read more …Infant with rare, incurable disease is first to successfully receive personalized gene therapy...

President Donald Trump and Republicans in the House of Representatives have put forward budget proposals[1] that would slash spending by hundreds of millions of dollars[2] over the next decade on several programs that support low-income U.S. families with children.

If those cuts are in the version of the 2026 budget that clears Congress, and Trump signs it into law, funding for early childhood education, support for grocery...

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