PUBLICATION

Zebrafish CCNF and FUS Mediate Stress-Specific Motor Responses

Authors
Aksoy, Y.A., Cole, A.J., Deng, W., Hesselson, D.
ID
ZDB-PUB-240313-15
Date
2024
Source
Cells   13(5): (Journal)
Registered Authors
Aksoy, Yagiz, Hesselson, Daniel
Keywords
ALS pathogenesis, Amyotrophic Lateral Sclerosis (ALS), CCNF gene, CRISPR, CRISPR/Cas9 genome editing, FUS gene, TALEN, TALEN-mediated genome editing, Zebrafish embryonic development, Zebrafish models, endoplasmic reticulum stress, genetic mutations in ALS, in vivo genome editing, motor function analysis, motor neuron development, neurodegenerative disease research, oxidative stress response, stress-induced motor impairment
MeSH Terms
  • Amyotrophic Lateral Sclerosis*/metabolism
  • Animals
  • Cyclins*/metabolism
  • Humans
  • Motor Neurons/pathology
  • Neurodegenerative Diseases*/metabolism
  • Proteins/metabolism
  • RNA-Binding Protein FUS*/genetics
  • RNA-Binding Protein FUS*/metabolism
  • Zebrafish*/metabolism
PubMed
38474336 Full text @ Cells
Abstract
Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease characterized by the degeneration of motor neurons. Mutations in the cyclin F (CCNF) and fused in sarcoma (FUS) genes have been associated with ALS pathology. In this study, we aimed to investigate the functional role of CCNF and FUS in ALS by using genome editing techniques to generate zebrafish models with genetic disruptions in these genes. Sequence comparisons showed significant homology between human and zebrafish CCNF and FUS proteins. We used CRISPR/Cas9 and TALEN-mediated genome editing to generate targeted disruptions in the zebrafish ccnf and fus genes. Ccnf-deficient zebrafish exhibited abnormal motor neuron development and axonal outgrowth, whereas Fus-deficient zebrafish did not exhibit developmental abnormalities or axonopathies in primary motor neurons. However, Fus-deficient zebrafish displayed motor impairments in response to oxidative and endoplasmic reticulum stress. The Ccnf-deficient zebrafish were only sensitized to endoplasmic reticulum stress, indicating that ALS genes have overlapping as well as unique cellular functions. These zebrafish models provide valuable platforms for studying the functional consequences of CCNF and FUS mutations in ALS pathogenesis. Furthermore, these zebrafish models expand the drug screening toolkit used to evaluate possible ALS treatments.
Genes / Markers
Figures
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Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping