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Colobomatous microphthalmia

ORPHA:98938Malformation syndromeAutosomal dominant, Autosomal recessiveAntenatal, Neonatal

Ассоциированные гены (14)

SHH
sonic hedgehog signaling molecule
Disease-causing germline mutation(s) in
OMIM: 600725
STRA6
signaling receptor and transporter of retinol STRA6
Disease-causing germline mutation(s) in
OMIM: 610745
VSX2
visual system homeobox 2
Disease-causing germline mutation(s) in
OMIM: 142993
GDF6
growth differentiation factor 6
Candidate gene tested in
OMIM: 601147
GDF3
growth differentiation factor 3
Disease-causing germline mutation(s) in
OMIM: 606522
ABCB6
ATP binding cassette subfamily B member 6 (LAN blood group)
Disease-causing germline mutation(s) in
OMIM: 605452
TENM3
teneurin transmembrane protein 3
Disease-causing germline mutation(s) in
OMIM: 610083
RBP4
retinol binding protein 4
Disease-causing germline mutation(s) in
OMIM: 180250
SOX2
SRY-box transcription factor 2
Disease-causing germline mutation(s) in
OMIM: 184429
SIX6
SIX homeobox 6
Candidate gene tested in
OMIM: 606326
OTX2
orthodenticle homeobox 2
Disease-causing germline mutation(s) in
OMIM: 600037
RAX
retina and anterior neural fold homeobox
Disease-causing germline mutation(s) in
OMIM: 601881
ALDH1A3
aldehyde dehydrogenase 1 family member A3
Disease-causing germline mutation(s) in
OMIM: 600463
PORCN
porcupine O-acyltransferase
Disease-causing germline mutation(s) in
OMIM: 300651

Эпидемиология (2)

Prevalence at birth
1-5 / 10 000
United Kingdom
Point prevalence
1-5 / 10 000
United Kingdom

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы