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Explore the latest stories and publications from NCATS and our partners, see upcoming events, or explore the variety of resources we provide to better understand translational science and our work.

News

Read the latest news from NCATS and its collaborators whose research is supported through the Center’s programs, or follow coverage of NCATS' translational research activities through national and local media.

Infant with Rare, Incurable Disease is First to Successfully Receive Personalized Gene Therapy Treatment

May 28, 2025 - NCATS News

  • Our Impact on Rare Diseases
  • Somatic Cell Genome Editing (SCGE)

NIH-supported gene-editing platform lays groundwork to rapidly develop treatments for other rare genetic diseases.

Personalized Gene Editing Saved a Baby, But the Tech’s Future Is Uncertain

May 28, 2025 - Media Coverage

  • Our Impact on Rare Diseases
  • Somatic Cell Genome Editing (SCGE)

Landmark Finding Could Pave the Way for Gene Editing Technology

May 19, 2025 - Media Coverage

  • Our Impact on Rare Diseases
  • Somatic Cell Genome Editing (SCGE)

Repairing a Broken Code of Life: In CRISPR Breakthrough, Rewriting the Misspelling of Infant’s DNA Cures Him of Rare Liver Disease

May 16, 2025 - Grantee/Partner News

  • Our Impact on Rare Diseases
  • Somatic Cell Genome Editing (SCGE)

Personalized CRISPR Therapy Successfully Treats Infant With Rare, Incurable CPS1 Deficiency

May 16, 2025 - Media Coverage

  • Our Impact on Rare Diseases
  • Somatic Cell Genome Editing (SCGE)

Baby Treated With First-Ever Personalized Gene Editing Treatment

May 16, 2025 - Media Coverage

  • Our Impact on Rare Diseases
  • Somatic Cell Genome Editing (SCGE)

Last updated on February 19, 2025