Gene-Editing breakthrough aims to fix immune cells in rare disease
NCT ID NCT06325709
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tests a new gene-editing approach for X-linked chronic granulomatous disease (X-CGD), a rare immune disorder. Researchers will collect stem cells from 10 adult male participants, use base editing to correct the genetic mutation, and return the cells after mild chemotherapy. The goal is to restore white blood cell function and reduce life-threatening infections. Participants will be followed for 15 years to monitor safety and effectiveness.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
base-edited autologous hematopoietic stem and progenitor cells
What this could lead to
If successful, this could provide a one-time gene repair that restores white blood cell function, reducing severe infections and hospitalizations for people with X-linked CGD.
What could go wrong
This is an early-phase trial with only 10 participants, so results may not apply broadly. The chemotherapy conditioning and long-term immune suppression carry risks, and the gene correction may not be durable or effective enough.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••