Gene therapy for children with an Artemis SCID immune problem
Treatments studied
Part of Genetic & congenital, Hormones & metabolism, Immune system & allergy clinical trials.
This early-phase study tests an “autologous” gene therapy, meaning they use the child’s own cells, adjusted with a working gene, to help restore immune function. It is for babies and children with Artemis-deficient SCID, aiming to improve T-cell immunity and reduce the need for lifelong infection risk management.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be at least 2 months old when treatment with busulfan begins
- Have a confirmed Artemis (DCLRE1C) gene change on both copies of the gene
- Have immune blood test results that fit typical or slightly “leaky” ART-SCID (your team will check CD3 counts and other specific T-cell measures)
- Have test results showing your immune cells respond weakly to a lab stimulation (PHA test result is very low)
- Have no healthy HLA-matched brother/sister who could donate bone marrow (this matters for newly diagnosed patients)
- Be able to safely get anesthesia, and have procedures like bone marrow harvest and a central line
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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