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46,XY partial gonadal dysgenesis

ORPHA:251510Malformation syndromeAutosomal dominant, Autosomal recessive, X-linked recessive, Y-linkedInfancy, Neonatal

Ассоциированные гены (11)

DHX37
DEAH-box helicase 37
Disease-causing germline mutation(s) in
OMIM: 617362
WT1
WT1 transcription factor
Candidate gene tested in
OMIM: 607102
SOX9
SRY-box transcription factor 9
Disease-causing germline mutation(s) in
OMIM: 608160
SRY
sex determining region Y
Disease-causing germline mutation(s) in
OMIM: 480000
WWOX
WW domain containing oxidoreductase
Disease-causing germline mutation(s) in
OMIM: 605131
ZFPM2
zinc finger protein, FOG family member 2
Disease-causing germline mutation(s) in
OMIM: 603693
GATA4
GATA binding protein 4
Disease-causing germline mutation(s) (loss of function) in
OMIM: 600576
NR0B1
nuclear receptor subfamily 0 group B member 1
Role in the phenotype of
OMIM: 300473
NR5A1
nuclear receptor subfamily 5 group A member 1
Disease-causing germline mutation(s) in
OMIM: 184757
MAP3K1
mitogen-activated protein kinase kinase kinase 1
Disease-causing germline mutation(s) in
OMIM: 600982
VAMP7
vesicle associated membrane protein 7
Role in the phenotype of
OMIM: 300053

Фенотипы (43)

Очень частый (80–99%)31
HP:0003251Male infertility
HP:0008214Decreased serum estradiol
HP:0008232Elevated circulating follicle stimulating hormone level
HP:0008665Clitoral hypertrophy
HP:0008726Hypoplasia of the vagina
HP:0008730Female external genitalia in individual with 46,XY karyotype
HP:0008734Decreased testicular size
HP:0008736Hypoplasia of penis
HP:0010464Streak ovary
HP:0011969Elevated circulating luteinizing hormone level
HP:0012244Abnormal sex determination
HP:0012870Vanishing testis
HP:0040171Decreased serum testosterone concentration
HP:0100779Urogenital sinus anomaly
HP:0000027Azoospermia
HP:0000045Abnormality of the scrotum
HP:0000047Hypospadias
HP:0000054Micropenis
HP:0000058Abnormality of the labia
HP:0000062Ambiguous genitalia
HP:0000133Gonadal dysgenesis
HP:0000142Abnormality of the vagina
HP:0000771Gynecomastia
HP:0000786Primary amenorrhea
HP:0000812Abnormal internal genitalia
HP:0000815Hypergonadotropic hypogonadism
HP:0000837Increased circulating gonadotropin level
HP:0000868Decreased fertility in females
HP:0000939Osteoporosis
HP:0002215Sparse axillary hair
HP:0002225Sparse pubic hair
Частый (30–79%)3
HP:0000028Cryptorchidism
HP:0000150Gonadoblastoma
HP:0000823Delayed puberty
Периодический (5–29%)6
HP:0000030Testicular gonadoblastoma
HP:0000149Ovarian gonadoblastoma
HP:0000846Adrenal insufficiency
HP:0002750Delayed skeletal maturation
HP:0008187Absence of secondary sex characteristics
HP:0008193Primary gonadal insufficiency
Очень редкий (1–4%)3
HP:0000100Nephrotic syndrome
HP:0002667Nephroblastoma
HP:0030680Abnormal cardiovascular system morphology

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