PUBLICATION

Hcfc1b, a zebrafish ortholog of HCFC1, regulates craniofacial development by modulating MMACHC expression

Authors
Quintana, A.M., Geiger, E.A., Achilly, N., Rosenblatt, D.S., Maclean, K.N., Stabler, S.P., Artinger, K.B., Appel, B., Shaikh, T.H.
ID
ZDB-PUB-141005-7
Date
2014
Source
Developmental Biology   396(1): 94-106 (Journal)
Registered Authors
Appel, Bruce, Artinger, Kristin Bruk
Keywords
Cobalamin, Craniofacial defects, Facial dysmorphia, HCFC1, MMACHC
MeSH Terms
  • Animals
  • Body Patterning/genetics
  • Branchial Region/physiology
  • Carrier Proteins/genetics
  • Carrier Proteins/physiology*
  • Cell Differentiation
  • Cell Movement
  • Chondrocytes/cytology
  • Craniofacial Abnormalities/genetics
  • Gene Expression Regulation, Developmental*
  • Gene Knockdown Techniques
  • Green Fluorescent Proteins/metabolism
  • Host Cell Factor C1/genetics
  • Host Cell Factor C1/physiology*
  • Humans
  • Mice, Transgenic
  • Mutation
  • Neural Crest/cytology
  • Neural Crest/physiology
  • Phenotype
  • Stem Cells/cytology
  • Vitamin B 12/metabolism
  • Zebrafish/genetics
  • Zebrafish Proteins/genetics
  • Zebrafish Proteins/physiology*
PubMed
25281006 Full text @ Dev. Biol.
Abstract
Mutations in HCFC1 (MIM300019), have been recently associated with cblX (MIM309541), an X-linked, recessive disorder characterized by multiple congenital anomalies including craniofacial abnormalities. HCFC1 is a transcriptional co-regulator that modulates the expression of numerous downstream target genes including MMACHC, but it is not clear how these HCFC1 targets play a role in the clinical manifestations of cblX. To begin to elucidate the mechanism by which HCFC1 modulates disease phenotypes, we have carried out loss of function analyses in the developing zebrafish. Of the two HCFC1 orthologs in zebrafish, hcfc1a and hcfc1b, the loss of hcfc1b specifically results in defects in craniofacial development. Subsequent analysis revealed that hcfc1b regulates cranial neural crest cell differentiation and proliferation within the posterior pharyngeal arches. Further, the hcfc1b-mediated craniofacial abnormalities were rescued by expression of human MMACHC, a downstream target of HCFC1 that is aberrantly expressed in cblX. Furthermore, we tested distinct human HCFC1 mutations for their role in craniofacial development and demonstrated variable effects on MMACHC expression in humans and craniofacial development in zebrafish. Notably, several individuals with mutations in either HCFC1 or MMACHC have been reported to have mild to moderate facial dysmorphia. Thus, our data demonstrates that HCFC1 plays a role in craniofacial development, which is in part mediated through the regulation of MMACHC expression.
Genes / Markers
Figures
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Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping