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Baby with rare disease given world-first personal CRISPR gene therapy

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An infant with a severe genetic condition has shown signs of improvement after receiving a gene-editing treatment tailored to his specific mutation Originally published   by Michael Le Page at newscientist.com on 15 May 2025 Baby KJ after a gene-editing infusion with researchers Rebecca Ahrens-Nicklas and Kiran Musunuru Children’s Hospital of Philadelphia A baby boy with a life-threatening genetic condition has become the first person to receive a bespoke CRISPR gene-editing treatment , giving a glimpse into what t he future of medicine might look like. It’s the first time anyone has been given a gene-editing treatment designed t o correct a disease-causing mutation found only in that individual, Rebecca Ahrens-Nicklas at the Children’s Hospital of Philadelphia, Pennsylvania , told a press briefing. “He’s showing some early signs of benefit,” she says, but it is too soon to tell how well the treatment worked. The researchers published the details as soon as possible...

Microbiome study finds bacteria in human gut rarely update their CRISPR defense systems

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Originally published by Anne Trafton, Massachusetts Institute of Technology , on December 23, 2024  A study from MIT biological engineers has yielded new insight into how bacteria in the gut microbiome adapt their CRISPR defenses as they encounter new threats. Credit: Donny Bliss, NIH Within the human digestive tract are trillions of bacteria from thousands of different species . These bacteria form communities that help digest food, fend off harmful microbes, and play many other roles in maintaining human health. These bacteria can be vulnerable to infection from viruses called bacteriophages . One of bacterial cells' most well-known defenses against these viruses is the CRISPR system , which evolved in bacteria to help t hem recognize and chop up viral DNA . A study from MIT biological engineers has yielded new insight into how bacteria in the gut microbiome adapt their CRISPR defenses as they encounter new threats . The researchers found that while bacteria grown in t...