Gene therapy offers hope for 'Bubble Boy' disease
NCT ID NCT01410019
First seen Nov 01, 2025 · Last updated Jun 15, 2026 · Updated 30 times
Summary
This trial tested a gene therapy to fix the faulty gene causing X-linked severe combined immunodeficiency (SCID-X1), a life-threatening condition where boys are born without a working immune system. Five boys who lacked a suitable bone marrow donor received a single treatment with a self-inactivating retroviral vector carrying the correct gene. The goal was to restore their immune defenses and allow them to live normally without lifelong medication.
Disclaimer
Read more
Show less
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.
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 SEVERE COMBINED IMMUNODEFICIENCY are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Hopital Necker
Paris, 75015, France
Conditions
Explore the condition pages connected to this study.