Severe combined immunodeficiency due to DCLRE1C deficiency
MONDO:0011225Severe combined immunodeficiency (SCID) due to DCLRE1C deficiency is a type of SCID characterized by severe and recurrent infections, diarrhea, failure to thrive, and cell sensitivity to ionizing radiation.
Also known as: DCLRE1C severe combined immunodeficiency (disease), SCID due to ARTEMIS deficiency, SCID due to DCLRE1C deficiency, SCID due to artemis deficiency, SCID, Athabascan type, SCID, Athabaskan type, severe combined immunodeficiency (disease) caused by mutation in DCLRE1C, severe combined immunodeficiency due to ARTEMIS deficiency
8 clinical trials for this condition and its sub-types.
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Gene therapy offers hope for babies with 'Bubble Boy' disease
⭐️ CURE ⭐️ Recruiting nowThis study tests a new gene therapy for children with a severe immune disorder called Artemis-SCID, where the body cannot fight infections. Doctors take the child's own blood stem cells, fix the faulty gene in a lab, and put the corrected cells back. The goal is to rebuild a work…
Phase: PHASE1, PHASE2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: ⭐️ CURE ⭐️
Last updated Jun 27, 2026 12:00 UTC
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New drug combo targets Hard-to-Treat cancers in early trial
Disease control Recruiting nowThis study tests a new drug called AZD4956, alone or with other cancer drugs, in people with advanced or spreading solid tumors that have a specific DNA repair defect (HRR deficiency). The main goals are to check safety, find the best dose, and see if the drug can shrink tumors. …
Phase: PHASE1, PHASE2 • Sponsor: AstraZeneca • Aim: Disease control
Last updated Aug 13, 2026 00:00 UTC
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Gene therapy offers new hope for babies with rare 'Bubble Boy' disease
Disease control Recruiting nowThis study tests a gene therapy for Artemis-deficient severe combined immunodeficiency (ART-SCID), a life-threatening immune disorder. It involves taking the patient's own blood stem cells, adding a working copy of the faulty gene, and infusing them back after mild chemotherapy. …
Phase: PHASE1, PHASE2 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated Jun 27, 2026 09:04 UTC