CRISPR gene editing frees thalassemia patients from lifelong transfusions
NCT ID NCT03655678
First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 27 times
Summary
This study tested a one-time treatment using CRISPR gene editing to fix a patient's own blood stem cells, aiming to help people with severe beta-thalassemia stop needing regular blood transfusions. 59 participants received the modified cells after a short course of chemotherapy. The goal was to see if the treatment could safely allow the body to produce enough healthy red blood cells on its own.
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 HEMOGLOBINOPATHIES 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
-
Ann & Robert Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
-
British Columbia Children's Hospital
Vancouver, Canada
-
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
-
Columbia University Medical Center
New York, New York, 10032, United States
-
Columbia University Medical Center (21+ years)
New York, New York, 10032, United States
-
Imperial College Healthcare NHS Trust, Hammersmith Hospital
London, United Kingdom
-
Lucile Packard Children's Hospital
Palo Alto, California, 94304, United States
-
Ospedale Pediatrico Bambino Gesù, IRCCS
Rome, Italy
-
Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine
Regensburg, Germany
-
The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers
Nashville, Tennessee, 37203, United States
-
The Hospital for Sick Children
Toronto, Canada
-
University College London Hospitals NHS Foundation Trust
London, United Kingdom
-
University Hospital Tübingen
Tübingen, Germany
-
Universitätsklinikum Düsseldorf Hospital Duesseldorf
Düsseldorf, Germany
Conditions
Explore the condition pages connected to this study.