PUBLICATION

FKRP-dependent glycosylation of fibronectin regulates muscle pathology in muscular dystrophy

Authors
Wood, A.J., Lin, C.H., Li, M., Nishtala, K., Alaei, S., Rossello, F., Sonntag, C., Hersey, L., Miles, L.B., Krisp, C., Dudczig, S., Fulcher, A.J., Gibertini, S., Conroy, P.J., Siegel, A., Mora, M., Jusuf, P., Packer, N.H., Currie, P.D.
ID
ZDB-PUB-210601-1
Date
2021
Source
Nature communications   12: 2951 (Journal)
Registered Authors
Alaei, Sara, Currie, Peter D., Dudczig, Stefanie, Jusuf, Patricia, Sonntag, Carmen
Keywords
none
MeSH Terms
  • Animals
  • Basement Membrane/metabolism
  • Basement Membrane/pathology
  • Cell Line
  • Disease Models, Animal
  • Fibronectins/metabolism*
  • Gene Knockout Techniques
  • Glycosylation
  • Glycosyltransferases/deficiency
  • Glycosyltransferases/genetics
  • Glycosyltransferases/metabolism*
  • Humans
  • Male
  • Muscle, Skeletal/metabolism
  • Muscle, Skeletal/pathology
  • Muscular Dystrophies/genetics
  • Muscular Dystrophies/metabolism*
  • Muscular Dystrophies/pathology*
  • Muscular Dystrophies, Limb-Girdle/genetics
  • Muscular Dystrophies, Limb-Girdle/metabolism
  • Muscular Dystrophies, Limb-Girdle/pathology
  • Muscular Dystrophy, Animal/genetics
  • Muscular Dystrophy, Animal/metabolism*
  • Muscular Dystrophy, Animal/pathology*
  • Mutation
  • Myoblasts, Skeletal/metabolism
  • Myoblasts, Skeletal/pathology
  • Pentosyltransferases/deficiency
  • Pentosyltransferases/genetics
  • Pentosyltransferases/metabolism*
  • Phenotype
  • Zebrafish
  • Zebrafish Proteins/deficiency
  • Zebrafish Proteins/genetics
  • Zebrafish Proteins/metabolism*
PubMed
34012031 Full text @ Nat. Commun.
Abstract
The muscular dystrophies encompass a broad range of pathologies with varied clinical outcomes. In the case of patients carrying defects in fukutin-related protein (FKRP), these diverse pathologies arise from mutations within the same gene. This is surprising as FKRP is a glycosyltransferase, whose only identified function is to transfer ribitol-5-phosphate to α-dystroglycan (α-DG). Although this modification is critical for extracellular matrix attachment, α-DG's glycosylation status relates poorly to disease severity, suggesting the existence of unidentified FKRP targets. Here we reveal that FKRP directs sialylation of fibronectin, a process essential for collagen recruitment to the muscle basement membrane. Thus, our results reveal that FKRP simultaneously regulates the two major muscle-ECM linkages essential for fibre survival, and establishes a new disease axis for the muscular dystrophies.
Genes / Markers
Figures
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Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping