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Retinitis pigmentosa

ORPHA:791DiseaseAutosomal dominant, Autosomal recessive, Mitochondrial inheritance, X-linked recessiveAdolescent, Adult, Childhood

Ассоциированные гены (88)

SCAPER
S-phase cyclin A associated protein in the ER
Disease-causing germline mutation(s) in
OMIM: 611611
IFT88
intraflagellar transport 88
Disease-causing germline mutation(s) in
OMIM: 600595
REEP6
receptor accessory protein 6
Disease-causing germline mutation(s) (loss of function) in
OMIM: 609346
ARHGEF18
Rho/Rac guanine nucleotide exchange factor 18
Disease-causing germline mutation(s) in
OMIM: 616432
BBS1
Bardet-Biedl syndrome 1
Disease-causing germline mutation(s) in
OMIM: 209901
KIAA1549
KIAA1549
Disease-causing germline mutation(s) in
OMIM: 613344
TMEM216
transmembrane protein 216
Disease-causing germline mutation(s) (loss of function) in
OMIM: 613277
IFT43
intraflagellar transport 43
Disease-causing germline mutation(s) in
OMIM: 614068
AHI1
Abelson helper integration site 1
Disease-causing germline mutation(s) in
OMIM: 608894
AGBL5
AGBL carboxypeptidase 5
Disease-causing germline mutation(s) in
OMIM: 615900
CC2D2A
coiled-coil and C2 domain containing 2A
Disease-causing germline mutation(s) in
OMIM: 612013
ABCA4
ATP binding cassette subfamily A member 4
Disease-causing germline mutation(s) in
OMIM: 601691
PRCD
photoreceptor disc component
Disease-causing germline mutation(s) in
OMIM: 610598
PRPF3
pre-mRNA processing factor 3
Disease-causing germline mutation(s) in
OMIM: 607301
PRPF31
pre-mRNA processing factor 31
Disease-causing germline mutation(s) in
OMIM: 606419
PRPF8
pre-mRNA processing factor 8
Disease-causing germline mutation(s) in
OMIM: 607300
PRPH2
peripherin 2
Disease-causing germline mutation(s) in
OMIM: 179605
RDH12
retinol dehydrogenase 12
Disease-causing germline mutation(s) in
OMIM: 608830
RGR
retinal G protein coupled receptor
Disease-causing germline mutation(s) in
OMIM: 600342
RHO
rhodopsin
Disease-causing germline mutation(s) in
OMIM: 180380
RLBP1
retinaldehyde binding protein 1
Disease-causing germline mutation(s) in
OMIM: 180090
ROM1
retinal outer segment membrane protein 1
Disease-causing germline mutation(s) in
OMIM: 180721
RP1
RP1 axonemal microtubule associated
Disease-causing germline mutation(s) in
OMIM: 603937
RP2
RP2 activator of ARL3 GTPase
Disease-causing germline mutation(s) in
OMIM: 300757
RP9
RP9 pre-mRNA splicing factor
Disease-causing germline mutation(s) in
OMIM: 607331
RPE65
retinoid isomerohydrolase RPE65
Disease-causing germline mutation(s) in
OMIM: 180069
RPGR
retinitis pigmentosa GTPase regulator
Disease-causing germline mutation(s) in
OMIM: 312610
SAG
S-antigen visual arrestin
Disease-causing germline mutation(s) in
OMIM: 181031
BBS2
Bardet-Biedl syndrome 2
Disease-causing germline mutation(s) in
OMIM: 606151
BEST1
bestrophin 1
Disease-causing germline mutation(s) in
OMIM: 607854
CA4
carbonic anhydrase 4
Disease-causing germline mutation(s) in
OMIM: 114760
CERKL
CERK like autophagy regulator
Disease-causing germline mutation(s) in
OMIM: 608381
TTC8
tetratricopeptide repeat domain 8
Disease-causing germline mutation(s) in
OMIM: 608132
TULP1
TUB like protein 1
Disease-causing germline mutation(s) in
OMIM: 602280
USH2A
usherin
Disease-causing germline mutation(s) in
OMIM: 608400
CLRN1
clarin 1
Disease-causing germline mutation(s) in
OMIM: 606397
CNGA1
cyclic nucleotide gated channel subunit alpha 1
Disease-causing germline mutation(s) in
OMIM: 123825
CRB1
crumbs cell polarity complex component 1
Disease-causing germline mutation(s) in
OMIM: 604210
CRX
cone-rod homeobox
Disease-causing germline mutation(s) in
OMIM: 602225
ARL6
ARF like GTPase 6
Disease-causing germline mutation(s) in
OMIM: 608845
FSCN2
fascin actin-bundling protein 2, retinal
Disease-causing germline mutation(s) in
OMIM: 607643
HGSNAT
heparan-alpha-glucosaminide N-acetyltransferase
Disease-causing germline mutation(s) in
OMIM: 610453
IMPDH1
inosine monophosphate dehydrogenase 1
Disease-causing germline mutation(s) in
OMIM: 146690
MERTK
MER proto-oncogene, tyrosine kinase
Disease-causing germline mutation(s) in
OMIM: 604705
NR2E3
nuclear receptor subfamily 2 group E member 3
Disease-causing germline mutation(s) in
OMIM: 604485
NRL
neural retina leucine zipper
Disease-causing germline mutation(s) in
OMIM: 162080
OFD1
OFD1 centriole and centriolar satellite protein
Disease-causing germline mutation(s) in
OMIM: 300170
PDE6A
phosphodiesterase 6A
Disease-causing germline mutation(s) in
OMIM: 180071
PDE6B
phosphodiesterase 6B
Disease-causing germline mutation(s) in
OMIM: 180072
SEMA4A
semaphorin 4A
Disease-causing germline mutation(s) in
OMIM: 607292
CNGB1
cyclic nucleotide gated channel subunit beta 1
Disease-causing germline mutation(s) in
OMIM: 600724
TOPORS
TOP1 binding arginine/serine rich protein, E3 ubiquitin ligase
Disease-causing germline mutation(s) in
OMIM: 609507
PROM1
prominin 1
Disease-causing germline mutation(s) in
OMIM: 604365
LRAT
lecithin retinol acyltransferase
Disease-causing germline mutation(s) in
OMIM: 604863
EYS
EGF-like photoreceptor maintenance factor
Disease-causing germline mutation(s) in
OMIM: 612424
IDH3B
isocitrate dehydrogenase (NAD(+)) 3 non-catalytic subunit beta
Disease-causing germline mutation(s) in
OMIM: 604526
SPATA7
spermatogenesis associated 7
Disease-causing germline mutation(s) in
OMIM: 609868
GUCA1B
guanylate cyclase activator 1B
Disease-causing germline mutation(s) in
OMIM: 602275
KLHL7
kelch like family member 7
Disease-causing germline mutation(s) in
OMIM: 611119
SNRNP200
small nuclear ribonucleoprotein U5 subunit 200
Disease-causing germline mutation(s) in
OMIM: 601664
PCARE
photoreceptor cilium actin regulator
Disease-causing germline mutation(s) in
OMIM: 613425
PDE6G
phosphodiesterase 6G
Disease-causing germline mutation(s) in
OMIM: 180073
IMPG2
interphotoreceptor matrix proteoglycan 2
Disease-causing germline mutation(s) in
OMIM: 607056
FAM161A
FAM161 centrosomal protein A
Disease-causing germline mutation(s) in
OMIM: 613596
ZNF513
zinc finger protein 513
Disease-causing germline mutation(s) in
OMIM: 613598
CDHR1
cadherin related family member 1
Disease-causing germline mutation(s) in
OMIM: 609502
DHDDS
dehydrodolichyl diphosphate synthase subunit
Disease-causing germline mutation(s) in
OMIM: 608172
PRPF6
pre-mRNA processing factor 6
Disease-causing germline mutation(s) in
OMIM: 613979
MAK
male germ cell associated kinase
Disease-causing germline mutation(s) in
OMIM: 154235
RBP3
retinol binding protein 3
Disease-causing germline mutation(s) in
OMIM: 180290
CFAP418
cilia and flagella associated protein 418
Disease-causing germline mutation(s) in
OMIM: 614477
IFT140
intraflagellar transport 140
Disease-causing germline mutation(s) in
OMIM: 614620
ZNF408
zinc finger protein 408
Disease-causing germline mutation(s) (loss of function) in
OMIM: 616454
ARL2BP
ARF like GTPase 2 binding protein
Disease-causing germline mutation(s) in
OMIM: 615407
NEK2
NIMA related kinase 2
Disease-causing germline mutation(s) (loss of function) in
OMIM: 604043
IFT172
intraflagellar transport 172
Disease-causing germline mutation(s) in
OMIM: 607386
TUB
TUB bipartite transcription factor
Disease-causing germline mutation(s) (loss of function) in
OMIM: 601197
KIZ
kizuna centrosomal protein
Disease-causing germline mutation(s) (loss of function) in
OMIM: 615757
SLC7A14
solute carrier family 7 member 14
Disease-causing germline mutation(s) in
OMIM: 615720
PRPF4
pre-mRNA splicing tri-snRNP complex factor PRPF4
Disease-causing germline mutation(s) (loss of function) in
OMIM: 607795
RP1L1
RP1 like 1
Disease-causing germline mutation(s) in
OMIM: 608581
AHR
aryl hydrocarbon receptor
Disease-causing germline mutation(s) in
OMIM: 600253
COQ8B
coenzyme Q8B
Disease-causing germline mutation(s) in
OMIM: 615567
IMPG1
interphotoreceptor matrix proteoglycan 1
Disease-causing germline mutation(s) in
OMIM: 602870
IDH3A
isocitrate dehydrogenase (NAD(+)) 3 catalytic subunit alpha
Candidate gene tested in
OMIM: 601149
DHX38
DEAH-box helicase 38
Disease-causing germline mutation(s) in
OMIM: 605584
ARL3
ARF like GTPase 3
Disease-causing germline mutation(s) in
OMIM: 604695
POMGNT1
protein O-linked mannose N-acetylglucosaminyltransferase 1 (beta 1,2-)
Disease-causing germline mutation(s) in
OMIM: 606822

Фенотипы (31)

Очень частый (80–99%)13
HP:0000405Conductive hearing impairment
HP:0000407Sensorineural hearing impairment
HP:0000505Visual impairment
HP:0000512Abnormal electroretinogram
HP:0000546Retinal degeneration
HP:0000613Photophobia
HP:0000618Blindness
HP:0000639Nystagmus
HP:0000648Optic atrophy
HP:0007675Progressive night blindness
HP:0007703Abnormality of retinal pigmentation
HP:0007737Bone spicule pigmentation of the retina
HP:0008046Abnormal retinal vascular morphology
Частый (30–79%)15
HP:0000501Glaucoma
HP:0000543Optic disc pallor
HP:0000563Keratoconus
HP:0000602Ophthalmoplegia
HP:0000662Nyctalopia
HP:0000842Hyperinsulinemia
HP:0001105Retinal atrophy
HP:0007787Posterior subcapsular cataract
HP:0007843Attenuation of retinal blood vessels
HP:0007994Peripheral visual field loss
HP:0011505Cystoid macular edema
HP:0012426Optic disc drusen
HP:0030466Abnormal full-field electroretinogram
HP:0030488Abnormal central response of multifocal electroretinogram
HP:0030610Photoreceptor outer segment loss on macular OCT
Периодический (5–29%)3
HP:0000551Color vision defect
HP:0007663Reduced visual acuity
HP:0030786Photopsia

Эпидемиология (9)

Point prevalence
1-5 / 10 000
Europe
Point prevalence
1-5 / 10 000
Worldwide
Point prevalence
1-5 / 10 000
Denmark
Point prevalence
1-5 / 10 000
Norway
Point prevalence
1-5 / 10 000
United States
Point prevalence
1-5 / 10 000
United Kingdom
Point prevalence
1-5 / 10 000
China
Point prevalence
1-5 / 10 000
Slovenia
Point prevalence
1-5 / 10 000
Korea, Republic of

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы