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Severe combined immunodeficiency due to DCLRE1C deficiency

ORPHA:275DiseaseAutosomal recessiveInfancy, Neonatal

Ассоциированные гены (1)

DCLRE1C
DNA cross-link repair 1C
Disease-causing germline mutation(s) in
OMIM: 605988

Фенотипы (28)

Очень частый (80–99%)2
HP:0005403Decreased total T cell count
HP:0010976Decreased total B cell count
Частый (30–79%)10
HP:0001890Autoimmune hemolytic anemia
HP:0002110Bronchiectasis
HP:0002718Recurrent bacterial infections
HP:0002720Decreased circulating IgA level
HP:0004315Decreased circulating IgG level
HP:0005390Recurrent opportunistic infections
HP:0031292Cutaneous abscess
HP:0200043Verrucae
HP:0200117Recurrent upper and lower respiratory tract infections
HP:6000070Cutaneous granuloma
Периодический (5–29%)15
HP:0000388Otitis media
HP:0000988Skin rash
HP:0001045Vitiligo
HP:0001508Failure to thrive
HP:0001973Autoimmune thrombocytopenia
HP:0002041Intractable diarrhea
HP:0002664Neoplasm
HP:0002960Autoimmunity
HP:0004429Recurrent viral infections
HP:0005681Juvenile rheumatoid arthritis
HP:0009098Chronic oral candidiasis
HP:0011107Recurrent aphthous stomatitis
HP:0011274Recurrent mycobacterial infections
HP:0031123Recurrent gastroenteritis
HP:0045080Decreased proportion of CD3-positive T cells
Очень редкий (1–4%)1
HP:0000872Hashimoto thyroiditis

Эпидемиология (1)

Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы