One-Shot gene therapy aims to free patients from lifelong transfusions
NCT ID NCT07489196
Summary
This study is testing a single dose of an experimental gene therapy called CS-101 in people with severe beta-thalassemia. The goal is to see if this one-time treatment is safe and can help patients produce enough of their own healthy hemoglobin, potentially freeing them from needing regular blood transfusions. The therapy works by editing a patient's own blood stem cells to restart production of a helpful fetal form of hemoglobin.
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 Β-THALASSEMIA MAJOR 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
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, China
Contact
Contact
-
Ruijin Hospital Shanghai JiaoTong University School of Medicine
Shanghai, Shanghai Municipality, China
Contact
Contact
-
The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, China
Contact
Contact
Conditions
Explore the condition pages connected to this study.