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Perrault syndrome type 1

ORPHA:642945Clinical subtype

Ассоциированные гены (9)

PRORP
protein only RNase P catalytic subunit
Disease-causing germline mutation(s) (loss of function) in
OMIM: 609947
CLPP
caseinolytic mitochondrial matrix peptidase proteolytic subunit
Disease-causing germline mutation(s) in
OMIM: 601119
ERAL1
Era like 12S mitochondrial rRNA chaperone 1
Disease-causing germline mutation(s) in
OMIM: 607435
HARS2
histidyl-tRNA synthetase 2, mitochondrial
Disease-causing germline mutation(s) in
OMIM: 600783
HSD17B4
hydroxysteroid 17-beta dehydrogenase 4
Disease-causing germline mutation(s) in
OMIM: 601860
LARS2
leucyl-tRNA synthetase 2, mitochondrial
Disease-causing germline mutation(s) in
OMIM: 604544
TWNK
twinkle mtDNA helicase
Disease-causing germline mutation(s) in
OMIM: 606075
GGPS1
geranylgeranyl diphosphate synthase 1
Disease-causing germline mutation(s) in
OMIM: 606982
RMND1
required for meiotic nuclear division 1 homolog
Disease-causing germline mutation(s) in
OMIM: 614917

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы