Gene editing for sickle cell disease in young adults
Treatments studied
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial uses gene editing to fix your own blood stem cells, so you can stop having severe sickle cell crises. It is for young adults with frequent pain episodes or who need regular blood transfusions.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 18 to 24 years old.
- You have sickle cell disease (type SS, SB0, or SB+).
- You have had 2 or more pain crises per year (or 8 or more blood transfusions in the last year).
- You have tried or cannot take hydroxyurea.
- You do not have a brother or sister who is a perfect match for a bone marrow transplant.
- You have never had a stroke.
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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