PUBLICATION

Effect of serotonin modulation on dystrophin-deficient zebrafish

Authors
Spinazzola, J.M., Lambert, M.R., Gibbs, D.E., Conner, J.R., Krikorian, G.L., Pareek, P., Rago, C., Kunkel, L.M.
ID
ZDB-PUB-200729-3
Date
2020
Source
Biology Open   9(8): (Journal)
Registered Authors
Kunkel, Louis M.
Keywords
Drug screening, Duchenne Muscular Dystrophy, Serotonin, Zebrafish
MeSH Terms
  • Animals
  • Birefringence
  • Drug Evaluation, Preclinical
  • Dystrophin/deficiency*
  • Dystrophin/metabolism
  • Monoamine Oxidase Inhibitors/pharmacology
  • Receptors, Serotonin
  • Selective Serotonin Reuptake Inhibitors/pharmacology
  • Serotonin/metabolism*
  • Serotonin Receptor Agonists/pharmacology
  • Survival Analysis
  • Zebrafish/metabolism*
PubMed
32718931 Full text @ Biol. Open
Abstract
Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disease caused by mutation of the dystrophin gene. Pharmacological therapies that function independently of dystrophin and complement strategies aimed at dystrophin restoration could significantly improve patient outcomes. Previous observations have suggested that serotonin pathway modulation ameliorates dystrophic pathology, and reapplication of serotonin modulators already used clinically would potentially hasten availability to DMD patients. In our study, we used dystrophin-deficient sapje and sapje-like zebrafish models of DMD for rapid and easy screening of several classes of serotonin pathway modulators as potential therapeutics. None of the candidate drugs tested significantly decreased the percentage of zebrafish exhibiting the dystrophic muscle phenotype in the short-term birefringence assay or lengthened the lifespan in the long-term survival assay. Although we did not identify an effective drug, we believe our data is of value to the DMD research community for future studies, and there is evidence that suggests serotonin modulation may still be a viable treatment strategy with further investigation. Given the widespread clinical use of selective serotonin reuptake inhibitors, tricyclic antidepressants, and reversible inhibitors of monoamine oxidase, their reapplication to DMD is an attractive strategy in the field's pursuit to identify pharmacological therapies to complement dystrophin restoration strategies.
Genes / Markers
Figures
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Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping