PUBLICATION

The effects of ush2a gene knockout on vesicle transport in photoreceptors

Authors
Han, S., Wang, Q., Cheng, M., Hu, Y., Liu, P., Hou, W., Liang, L.
ID
ZDB-PUB-231010-56
Date
2023
Source
Gene   892: 147885 (Journal)
Registered Authors
Keywords
Pathogenesis, Retinitis pigmentosa, USH2A, Vesicle transport, Zebrafish
MeSH Terms
  • Animals
  • Extracellular Matrix Proteins/genetics
  • Extracellular Matrix Proteins/metabolism
  • Gene Knockout Techniques
  • Mutation
  • Photoreceptor Cells/metabolism
  • Retinitis Pigmentosa*/genetics
  • Usher Syndromes*/genetics
  • Zebrafish/genetics
  • Zebrafish/metabolism
PubMed
37813209 Full text @ Gene
Abstract
USH2A (Usher syndrome type 2A) gene mutations are the predominant cause of Usher syndrome type 2, characterized by sensorineural hearing loss and retinitis pigmentosa (RP), and also significant contributors to non-syndromic RP. To date, there is a lack of definitive therapeutic interventions to mitigate the associated disorders caused by USH2A mutations, and the precise pathogenic mechanisms underlying their onset remain unclear. In the present study, we utilized the ush2a knockout zebrafish model to investigate the pathological mechanisms of RP. In late-stage ush2a-/- zebrafish, the outer segments of rods displayed shortened length and decreased number. Anomalous vesicle accumulation was observed at the junction between the inner and outer segments, accompanied by reduced expression and structural damage of actin filaments in the photoreceptor cells. Furthermore, we discovered that Rab8 expression was downregulated and exhibited aberrant localization in ush2a-/- zebrafish. Additionally, we identified an interaction between USH2A and Rab8. Therefore, the knockout of ush2a may potentially affect vesicle transport through the regulation of Rab8, providing a novel target for maintaining the survival of photoreceptor cells. These findings also contribute to our understanding of the potential molecular pathogenesis underlying RP caused by USH2A gene mutations.
Genes / Markers
Figures
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Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping