Cure My Disease

Don't miss your cure!

 Log in / Sign up

Gene-Editing breakthrough aims to fix immune cells in rare disease

NCT ID NCT06325709

Recruiting now Disease control Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) Source: ClinicalTrials.gov ↗

First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 36 times

Summary

This early-stage trial tests a new gene-editing approach for X-linked chronic granulomatous disease (CGD), a rare immune disorder. Researchers will take stem cells from 10 adult males, fix a specific genetic mutation using base editing, and return the corrected cells after mild chemotherapy. The goal is to restore white blood cell function and reduce severe infections, but participants must take lifelong medication (sirolimus) to prevent immune reactions, so this is a disease-control strategy, not a cure.

Disclaimer Read more

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for X-LINKED CHRONIC GRANULOMATOUS DISEASE are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.

CHRONIC GRANULOMATOUS DISEASE (CGD) X-LINKED CHRONIC GRANULOMATOUS DISEASE