Granulomatous disease, chronic, autosomal recessive, cytochrome b-positive, type 1
MONDO:0009309Any chronic granulomatous disease in which the cause of the disease is a mutation in the NCF1 gene.
Also known as: NCF1 chronic granulomatous disease, chronic granulomatous disease 1, autosomal recessive, chronic granulomatous disease caused by mutation in NCF1, granulomatous disease, chronic, autosomal recessive, cytochrome b-positive, type 1, CDG1, CGD, autosomal recessive cytochrome B-positive, type 1, Ncf1, deficiency of, Soc2, deficiency of
45 clinical trials for this condition and its sub-types, 3 tagged with Granulomatous disease, chronic, autosomal recessive, cytochrome b-positive, type 1 itself.
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New gene therapy aims to fix immune system in rare disease
Disease control By invitation onlyThis study tests a new gene therapy for people with chronic granulomatous disease (CGD), a genetic disorder that weakens the immune system and makes them prone to serious infections. The treatment takes the patient's own blood stem cells, adds a missing gene in the lab, and puts …
Phase 1/2 • Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Disease control
Last updated Sep 17, 2026 00:00 UTC
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Groundbreaking gene therapy aims to fix immune system in rare disease
Disease control By invitation onlyThis early-stage trial tests a new gene therapy (PM359) for people with a rare immune disorder called chronic granulomatous disease (CGD). The treatment takes a patient's own blood stem cells, fixes a specific genetic error using a precise editing tool called prime editing, and r…
Phase 1/2 • Sponsor: Prime Medicine, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC