At 26, Shemiah Marcus-McDonald expected her first pregnancy to be full of changes. What she didn’t…
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Desperately ill baby healed with personalized gene therapy, doctors say
A baby born with a rare and dangerous genetic disease is growing and thriving after getting…
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Baby with rare disease given world-first personal CRISPR gene therapy
Baby KJ after a gene-editing infusion with researchers Rebecca Ahrens-Nicklas and Kiran Musunuru Children’s Hospital of…
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Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment
News Release Thursday, May 15, 2025 NIH-supported gene-editing platform lays groundwork to rapidly develop treatments…
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