PUBLICATION

Zebrafish gene knockdowns imply roles for human YWHAG in infantile spasms and cardiomegaly

Authors
Komoike, Y., Fujii, K., Nishimura, A., Hiraki, Y., Hayashidani, M., Shimojima, K., Nishizawa, T., Higashi, K., Yasukawa, K., Saitsu, H., Miyake, N., Mizuguchi, T., Matsumoto, N., Osawa, M., Kohno, Y., Higashinakagawa, T., and Yamamoto, T.
ID
ZDB-PUB-100211-32
Date
2010
Source
Genesis (New York, N.Y. : 2000)   48(4): 233-243 (Journal)
Registered Authors
Keywords
Williams-Beuren syndrome (WBS), infantile spasms, cardiomegaly, YWHAG, HIP1, developmental delay, array comparative genomic hybridization (aCGH), fluorescent in situ hybridization (FISH), 7q11.23, microdeletion
MeSH Terms
  • 14-3-3 Proteins/genetics*
  • 14-3-3 Proteins/metabolism
  • Animals
  • Brain/metabolism
  • Brain/pathology
  • Calmodulin/genetics
  • Calmodulin/metabolism
  • Cardiomegaly/genetics*
  • Cardiomegaly/metabolism
  • Cardiomegaly/pathology
  • Chromosome Deletion
  • Chromosomes, Human, Pair 7/genetics
  • Chromosomes, Human, Pair 7/metabolism
  • Comparative Genomic Hybridization
  • DNA-Binding Proteins/genetics
  • DNA-Binding Proteins/metabolism
  • Female
  • Gene Knockdown Techniques
  • Humans
  • Infant
  • Infant, Newborn
  • Male
  • Myocardium/metabolism
  • Myocardium/pathology
  • Oligonucleotide Array Sequence Analysis
  • Organ Size/genetics
  • Spasms, Infantile/genetics*
  • Spasms, Infantile/metabolism
  • Spasms, Infantile/pathology
  • Telomere/genetics
  • Telomere/metabolism
  • Telomere/pathology
  • Williams Syndrome/genetics*
  • Williams Syndrome/metabolism
  • Williams Syndrome/pathology
  • Zebrafish/genetics*
  • Zebrafish/metabolism
  • Zebrafish Proteins/genetics*
  • Zebrafish Proteins/metabolism
PubMed
20146355 Full text @ Genesis
Abstract
Williams-Beuren syndrome (WBS) is a neurodevelopmental disorder presenting with an elfin-like face, supravalvular aortic stenosis, a specific cognitive-behavioral profile, and infantile hypercalcemia. We encountered two WBS patients presenting with infantile spasms, which is extremely rare in WBS. Array comparative genomic hybridization (aCGH) and fluorescent in situ hybridization (FISH) analyses revealed atypical 5.7-Mb and 4.1-Mb deletions at 7q11.23 in the two patients, including the WBS critical region and expanding into the proximal side and the telomeric side, respectively. On the proximal side, AUTS2 and CALN1 may contribute to the phenotype. On the telomeric side, there are two candidate genes HIP1 and YWHAG. Because detailed information of them was unavailable, we investigated their functions using gene knockdowns of zebrafish. When zebrafish ywhag1 was knocked down, reduced brain size and increased diameter of the heart tube were observed, indicating that the infantile spasms and cardiomegaly seen in the patient with the telomeric deletion may be derived from haploinsufficiency of YWHAG.
Genes / Markers
Figures
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Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping