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Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy | MicrobiomeTrials
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Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy ENROLLING_BY_INVITATION PHASE1, PHASE2 INTERVENTIONAL
Read the full scientific description See full eligibility requirements Trial Details Start: 2025-05-09Est. Completion: 2034-12-31
Enrollment target: 18 participantsLocations National Institutes of Health Clinical Center, Bethesda, Maryland, United States Other microbiome trials you might be interested in X-linked Severe Combined Immunodeficiency (XSCID)
This is a Phase I/II non-randomized clinical trial of ex vivo hematopoietic stem cell (HSC) gene transfer treatment for …
Bethesda, United States Est. completion Dec 31, 2032
Common Variable Immunodeficiency (CVID) Primary Immune Regulatory Disorder Immune Dysregulation DiGeorge Syndrome STAT 1 Gain of Function STAT 3 Gain of Function
Hypomorphic RAG1 Deficiency
CD40 Ligand Deficiency
Mendelian Susceptibility to Mycobacterial Disease
GATA2 Associated Immunodeficiency
CD40 Deficiency
Hypomorphic RAG2 Deficiency
Immune Dysregulation Polyendocrinopathy Enteropathy X-Linked Syndrome
Omenn Syndrome
Chronic Granulomatous Disease
This is a research protocol that will examine Hematopoietic Stem Cell Transplantation (HSCT) using a reduced conditionin…
Pittsburgh, United States Est. completion May 4, 2030