Scientists test One-Shot genetic cure for devastating blood disease
NCT ID NCT02247843
Summary
This early-stage study tested a potential one-time treatment for adults with severe sickle cell disease. Doctors collected a patient's own blood stem cells, used a modified virus to insert a healthy gene into them, and then gave them back to the patient. The goal was to see if this process was safe and could allow the body to produce healthy red blood cells for life, potentially curing the disease.
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 SICKLE CELL DISEASE 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
-
University of California, Los Angeles (UCLA)
Los Angeles, California, 90095, United States
Conditions
Explore the condition pages connected to this study.