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Meckel syndrome

ORPHA:564Malformation syndromeAutosomal recessiveAntenatal

Ассоциированные гены (17)

TXNDC15
thioredoxin domain containing 15
Disease-causing germline mutation(s) (loss of function) in
OMIM: 617778
TMEM107
transmembrane protein 107
Disease-causing germline mutation(s) (loss of function) in
OMIM: 616183
TCTN1
tectonic family member 1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 609863
TMEM237
transmembrane protein 237
Disease-causing germline mutation(s) (loss of function) in
OMIM: 614423
RPGRIP1
RPGR interacting protein 1
Candidate gene tested in
OMIM: 605446
CEP290
centrosomal protein 290
Disease-causing germline mutation(s) in
OMIM: 610142
TMEM67
transmembrane protein 67
Disease-causing germline mutation(s) in
OMIM: 609884
MKS1
MKS transition zone complex subunit 1
Disease-causing germline mutation(s) in
OMIM: 609883
RPGRIP1L
RPGRIP1 like
Disease-causing germline mutation(s) in
OMIM: 610937
CC2D2A
coiled-coil and C2 domain containing 2A
Disease-causing germline mutation(s) in
OMIM: 612013
TMEM216
transmembrane protein 216
Disease-causing germline mutation(s) in
OMIM: 613277
TCTN3
tectonic family member 3
Disease-causing germline mutation(s) (loss of function) in
OMIM: 613847
TCTN2
tectonic family member 2
Disease-causing germline mutation(s) in
OMIM: 613846
B9D1
B9 domain containing 1
Disease-causing germline mutation(s) in
OMIM: 614144
B9D2
B9 domain containing 2
Disease-causing germline mutation(s) in
OMIM: 611951
TMEM231
transmembrane protein 231
Disease-causing germline mutation(s) (loss of function) in
OMIM: 614949
CSPP1
centrosome and spindle pole associated protein 1
Disease-causing germline mutation(s) in
OMIM: 611654

Фенотипы (45)

Очень частый (80–99%)6
HP:0000003Multicystic kidney dysplasia
HP:0000252Microcephaly
HP:0001162Postaxial hand polydactyly
HP:0001830Postaxial foot polydactyly
HP:0002084Encephalocele
HP:0002612Congenital hepatic fibrosis
Частый (30–79%)19
HP:0000358Posteriorly rotated ears
HP:0000028Cryptorchidism
HP:0000062Ambiguous genitalia
HP:0000175Cleft palate
HP:0000293Full cheeks
HP:0000316Hypertelorism
HP:0000340Sloping forehead
HP:0000347Micrognathia
HP:0000457Depressed nasal ridge
HP:0000482Microcornea
HP:0000518Cataract
HP:0000532Chorioretinal abnormality
HP:0000568Microphthalmia
HP:0000647Sclerocornea
HP:0000648Optic atrophy
HP:0001562Oligohydramnios
HP:0001883Talipes
HP:0006870Lobar holoprosencephaly
HP:0008053Aplasia/Hypoplasia of the iris
Периодический (5–29%)20
HP:0000037Male pseudohermaphroditism
HP:0000068Urethral atresia
HP:0000073Ureteral duplication
HP:0000221Furrowed tongue
HP:0000238Hydrocephalus
HP:0000528Anophthalmia
HP:0001177Preaxial hand polydactyly
HP:0001305Dandy-Walker malformation
HP:0001696Situs inversus totalis
HP:0001737Pancreatic cysts
HP:0001746Asplenia
HP:0001747Accessory spleen
HP:0002323Anencephaly
HP:0006487Bowing of the long bones
HP:0006706Cystic liver disease
HP:0007370Aplasia/Hypoplasia of the corpus callosum
HP:0010295Aplasia/Hypoplasia of the tongue
HP:0010459True hermaphroditism
HP:0030680Abnormal cardiovascular system morphology
HP:0100732Pancreatic fibrosis

Эпидемиология (6)

Prevalence at birth
1-9 / 100 000
France
Point prevalence
<1 / 1 000 000
France
Prevalence at birth
1-5 / 10 000
Finland
Prevalence at birth
1-9 / 100 000
Worldwide
Point prevalence
<1 / 1 000 000
Worldwide
Prevalence at birth
1-9 / 100 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы