Gene editing for severe sickle cell or thalassemia
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a new gene therapy that edits your own blood stem cells to help them make more fetal hemoglobin, which can prevent sickling and reduce the need for transfusions. It is for people with severe sickle cell disease or beta thalassemia who do not have a matched sibling donor.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have sickle cell disease (certain types) or transfusion-dependent beta thalassemia.
- You are between 13 and 40 years old.
- Your disease is serious: for sickle cell you have had frequent pain crises or lung problems; for thalassemia you need many blood transfusions.
- You have enough healthy blood cells and your organs (kidneys, lungs, heart, liver) are working well.
- You have tried hydroxyurea (for sickle cell) and it didn’t help enough, or you can’t take it.
- You are willing to come back for checkups for 15 years after treatment.
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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