Gene therapy for transfusion-dependent beta-thalassemia
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a new gene therapy for people with beta-thalassemia who need regular blood transfusions. It uses a virus to deliver a working gene into your own stem cells, then those cells are put back into your body to help your body make healthy red blood cells.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 3 and 35 years old.
- You have beta-thalassemia that requires regular blood transfusions (about 8 or more per year).
- You are able to have a stem cell transplant (using your own cells) and are healthy enough for the procedure.
- You have not had a stem cell transplant or gene therapy before.
- You do not have a matched donor available for a standard stem cell transplant.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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This trial tests a new treatment that uses your own blood stem cells, edited with CRISPR technology, to reduce or eliminate the need for blood transfusions in people with beta-thalassemia.
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