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Autosomal dominant polycystic kidney disease

ORPHA:730DiseaseAutosomal dominantAdolescent, Adult, Childhood

Ассоциированные гены (9)

GANAB
glucosidase II alpha subunit
Disease-causing germline mutation(s) in
OMIM: 104160
NEK8
NIMA related kinase 8
Disease-causing germline mutation(s) in
OMIM: 609799
ALG9
ALG9 alpha-1,2-mannosyltransferase
Disease-causing germline mutation(s) (loss of function) in
OMIM: 606941
DNAJB11
DnaJ heat shock protein family (Hsp40) member B11
Disease-causing germline mutation(s) (loss of function) in
OMIM: 611341
ALG5
ALG5 dolichyl-phosphate beta-glucosyltransferase
Disease-causing germline mutation(s) in
OMIM: 604565
PKD1
polycystin 1, transient receptor potential channel interacting
Disease-causing germline mutation(s) in
OMIM: 601313
PKD2
polycystin 2, transient receptor potential cation channel
Disease-causing germline mutation(s) in
OMIM: 173910
BICC1
BicC family RNA binding protein 1
Candidate gene tested in
OMIM: 614295
IFT140
intraflagellar transport 140
Disease-causing germline mutation(s) (loss of function) in
OMIM: 614620

Фенотипы (26)

Очень частый (80–99%)5
HP:0000083Renal insufficiency
HP:0000107Renal cyst
HP:0001407Hepatic cysts
HP:0003259Elevated circulating creatinine concentration
HP:0012213Decreased glomerular filtration rate
Частый (30–79%)7
HP:0000790Hematuria
HP:0000822Hypertension
HP:0003774Stage 5 chronic kidney disease
HP:0012591Abnormal urinary electrolyte concentration
HP:0012592Albuminuria
HP:0012622Chronic kidney disease
HP:0030157Flank pain
Периодический (5–29%)13
HP:0000010Recurrent urinary tract infections
HP:0000105Enlarged kidney
HP:0000791Uric acid nephrolithiasis
HP:0001634Mitral valve prolapse
HP:0001737Pancreatic cysts
HP:0002616Aortic root aneurysm
HP:0004944Dilatation of the cerebral artery
HP:0006557Polycystic liver disease
HP:0008672Calcium oxalate nephrolithiasis
HP:0011004Abnormal systemic arterial morphology
HP:0012207Reduced sperm motility
HP:0012330Pyelonephritis
HP:0100702Arachnoid cyst
Очень редкий (1–4%)1
HP:0011760Pituitary growth hormone cell adenoma

Эпидемиология (1)

Point prevalence
1-5 / 10 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы