Doctors test gene fix for 'Bubble Boy' disease in infants
NCT ID NCT01512888
Summary
This study tested a new gene therapy method for infants with SCID-X1, a severe genetic immune disorder. Doctors collected the baby's own bone marrow cells, inserted a corrected gene copy in the lab, and then returned the cells to the baby. The goal was to see if this procedure was safe and could help the baby develop a working immune system, especially for those without a matched sibling donor for a standard transplant.
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 SEVERE COMBINED IMMUNODEFICIENCY DISEASE, X-LINKED 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
-
Seattle Children's Research Institute
Seattle, Washington, 98101, United States
-
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
-
University of California-San Francisco
San Francisco, California, 94158, United States
Conditions
Explore the condition pages connected to this study.