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News

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.

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)

Infant With Rare Incurable Disease Becomes First to Receive Successful Personalized Gene Therapy

May 15, 2025 - Media Coverage

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

Infant With Rare, Incurable Disease Is First to Successfully Receive Personalized Gene Therapy Treatment

May 15, 2025 - Grantee/Partner News

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

Researchers Identify Pathway Responsible for Calciphylaxis, a Rare and Serious Condition

April 23, 2025 - Grantee/Partner News

  • Clinical and Translational Science Awards (CTSA) Program

Last updated on September 8, 2025