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New gene therapy could help people with rare immune disease fight infections

NCT ID NCT06253507

First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 33 times

Summary

This study tests a new gene therapy for people with a rare genetic disorder called chronic granulomatous disease (CGD), which makes them prone to severe infections. The treatment involves taking the patient's own stem cells, adding a missing gene in the lab, and returning them to the body. The goal is to help the immune system work better and reduce infections. Up to 10 people aged 3 and older will participate and be followed for 3 years.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States

Conditions

Explore the condition pages connected to this study.