PUBLICATION

Pharmacological restoration of visual function in a zebrafish model of von-Hippel Lindau disease

Authors
Ward, R., Ali, Z., Slater, K., Reynolds, A.L., Jensen, L.D., Kennedy, B.N.
ID
ZDB-PUB-190303-9
Date
2019
Source
Developmental Biology   457(2): 226-234 (Journal)
Registered Authors
Kennedy, Breandan N., Reynolds, Alison
Keywords
Retinal neovascularisation, Sunitinib malate, VHL, Von Hippel-Lindau, Zebrafish
MeSH Terms
  • Animals
  • Antineoplastic Agents/pharmacology
  • Blindness/genetics
  • Disease Models, Animal
  • Eye/blood supply*
  • Eye/embryology
  • Hemangioblastoma/genetics
  • Retina/embryology*
  • Sunitinib/pharmacology
  • Tumor Suppressor Proteins/genetics*
  • Vision, Ocular/genetics
  • Zebrafish/genetics*
  • Zebrafish Proteins/genetics*
  • von Hippel-Lindau Disease/genetics*
  • von Hippel-Lindau Disease/pathology
  • von Hippel-Lindau Disease/prevention & control
PubMed
30825427 Full text @ Dev. Biol.
Abstract
Von Hippel-Lindau (VHL) syndrome is rare, autosomal dominant disorder, characterised by hypervascularised tumour formation in multiple organ systems. Vision loss associated with retinal capillary hemangioblastomas remains one of the earliest complications of VHL disease. The mortality of Vhl-/- mice in utero restricted modelling of VHL disease in this mammalian model. Zebrafish harbouring a recessive germline mutation in the vhl gene represent a viable, alterative vertebrate model to investigate associated ocular loss-of-function phenotypes. Previous studies reported neovascularisation of the brain, eye and trunk together with oedema in the vhl-/- zebrafish eye. In this study, we demonstrate vhl-/- zebrafish almost entirely lack visual function. Furthermore, hyaloid vasculature networks in the vhl-/- eye are improperly formed and this phenotype is concomitant with development of an ectopic intraretinal vasculature. Sunitinib, a multi tyrosine kinase inhibitor, market authorised for cancer, reversed the ocular behavioural and morphological phenotypes observed in vhl-/- zebrafish. We conclude that the zebrafish vhl gene contributes to an endogenous molecular barrier that prevents development of intraretinal vasculature, and that pharmacological intervention with sunitinib can improve visual function and hyaloid vessel patterning while reducing abnormally formed ectopic intraretinal vessels in vhl-/- zebrafish.
Genes / Markers
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Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping