Gene therapy for ADA-SCID using a new delivery vector
Part of Genetic & congenital, Immune system & allergy clinical trials.
This study tests a new gene therapy that adds a working copy of the missing ADA gene to your own blood stem cells, helping your immune system fight infections. It is for people with ADA-SCID who have not had a prior transplant.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have the genetic form of ADA-SCID (a severe immune system problem caused by a missing enzyme).
- You have low T-cell counts or your immune cells do not respond normally to tests.
- You have had serious infections (like pneumonia, prolonged diarrhea, or certain viruses).
- You have not had a stem cell transplant before.
- You are expected to live at least 2 more months and are HIV-negative.
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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