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Cone rod dystrophy

ORPHA:1872DiseaseAutosomal dominant, Autosomal recessive, X-linked recessiveAdolescent, Adult, Childhood

Ассоциированные гены (32)

RPGR
retinitis pigmentosa GTPase regulator
Disease-causing germline mutation(s) in
OMIM: 312610
PRPH2
peripherin 2
Disease-causing germline mutation(s) in
OMIM: 179605
CFAP410
cilia and flagella associated protein 410
Disease-causing germline mutation(s) in
OMIM: 603191
PITPNM3
PITPNM family member 3
Disease-causing germline mutation(s) in
OMIM: 608921
OPN1MW
opsin 1, medium wave sensitive
Disease-causing germline mutation(s) in
OMIM: 300821
CRX
cone-rod homeobox
Disease-causing germline mutation(s) in
OMIM: 602225
NMNAT1
nicotinamide nucleotide adenylyltransferase 1
Disease-causing germline mutation(s) in
OMIM: 608700
CFAP418
cilia and flagella associated protein 418
Disease-causing germline mutation(s) in
OMIM: 614477
CDHR1
cadherin related family member 1
Disease-causing germline mutation(s) in
OMIM: 609502
MFSD8
major facilitator superfamily domain containing 8
Disease-causing germline mutation(s) in
OMIM: 611124
ABCA4
ATP binding cassette subfamily A member 4
Disease-causing germline mutation(s) in
OMIM: 601691
RIMS1
regulating synaptic membrane exocytosis 1
Disease-causing germline mutation(s) in
OMIM: 606629
RPGRIP1
RPGR interacting protein 1
Disease-causing germline mutation(s) in
OMIM: 605446
CACNA1F
calcium voltage-gated channel subunit alpha1 F
Disease-causing germline mutation(s) in
OMIM: 300110
CNGA3
cyclic nucleotide gated channel subunit alpha 3
Disease-causing germline mutation(s) in
OMIM: 600053
AIPL1
AIP like 1 HSP90 co-chaperone
Candidate gene tested in
OMIM: 604392
GUCA1A
guanylate cyclase activator 1A
Disease-causing germline mutation(s) in
OMIM: 600364
GUCY2D
guanylate cyclase 2D, retinal
Disease-causing germline mutation(s) in
OMIM: 600179
OPN1LW
opsin 1, long wave sensitive
Disease-causing germline mutation(s) in
OMIM: 300822
RAX2
retina and anterior neural fold homeobox 2
Disease-causing germline mutation(s) in
OMIM: 610362
SEMA4A
semaphorin 4A
Disease-causing germline mutation(s) in
OMIM: 607292
PROM1
prominin 1
Disease-causing germline mutation(s) in
OMIM: 604365
CACNA2D4
calcium voltage-gated channel auxiliary subunit alpha2delta 4
Disease-causing germline mutation(s) in
OMIM: 608171
ADAM9
ADAM metallopeptidase domain 9
Disease-causing germline mutation(s) in
OMIM: 602713
UNC119
unc-119 lipid binding chaperone
Disease-causing germline mutation(s) in
OMIM: 604011
RAB28
RAB28, member RAS oncogene family
Disease-causing germline mutation(s) in
OMIM: 612994
POC1B
POC1 centriolar protein B
Disease-causing germline mutation(s) (loss of function) in
OMIM: 614784
DRAM2
DNA damage regulated autophagy modulator 2
Disease-causing germline mutation(s) (loss of function) in
OMIM: 613360
TTLL5
tubulin tyrosine ligase like 5
Disease-causing germline mutation(s) (loss of function) in
OMIM: 612268
TLCD3B
TLC domain containing 3B
Disease-causing germline mutation(s) (loss of function) in
OMIM: 615175
UBAP1L
ubiquitin associated protein 1 like
Disease-causing germline mutation(s) in
ATF6
activating transcription factor 6
Disease-causing germline mutation(s) in
OMIM: 605537

Фенотипы (15)

Очень частый (80–99%)3
HP:0000613Photophobia
HP:0000662Nyctalopia
HP:0007703Abnormality of retinal pigmentation
Частый (30–79%)9
HP:0000529Progressive visual loss
HP:0000543Optic disc pallor
HP:0000551Color vision defect
HP:0000603Central scotoma
HP:0001105Retinal atrophy
HP:0007641Dyschromatopsia
HP:0007737Bone spicule pigmentation of the retina
HP:0007843Attenuation of retinal blood vessels
HP:0030466Abnormal full-field electroretinogram
Периодический (5–29%)3
HP:0000505Visual impairment
HP:0000639Nystagmus
HP:0012508Metamorphopsia

Эпидемиология (1)

Point prevalence
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