Document Detail


Cardiovascular defects in a mouse model of HOXA1 syndrome.
MedLine Citation:
PMID:  21940751     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
Congenital heart disease is one of the most common human birth defects, yet many genes and pathways regulating heart development remain unknown. A recent study in humans revealed that mutations in a single Hox gene, HOXA1 (Athabascan Brainstem Dysgenesis Syndrome, Bosley-Salih-Alorainy Syndrome), can cause severe cardiovascular malformations, some of which are lethal without surgical intervention. Since the discovery of the human syndromes, there have been no reports of any Hox mouse mutants with cardiac defects, hampering studies to explore the developmental causes of the human disease. In this study, we identify severe cardiovascular malformations in a Hox mouse model, which mimic the congenital heart defects in HOXA1 syndrome patients. Hoxa1 null mice show defects such as interrupted aortic arch, aberrant subclavian artery and Tetralogy of Fallot, demonstrating that Hoxa1 is required for patterning of the great arteries and outflow tract of the heart. We show that during early embryogenesis, Hoxa1 is expressed in precursors of cardiac neural crest cells (NCCs), which populate the heart. We further demonstrate that Hoxa1 acts upstream of several genes, important for neural crest specification. Thus, our data allow us to suggest a model in which Hoxa1 regulates heart development through its influence on cardiac NCCs, providing insight into the mechanisms underlying the human disease.
Authors:
Nadja Makki; Mario R Capecchi
Publication Detail:
Type:  Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't     Date:  2011-09-22
Journal Detail:
Title:  Human molecular genetics     Volume:  21     ISSN:  1460-2083     ISO Abbreviation:  Hum. Mol. Genet.     Publication Date:  2012 Jan 
Date Detail:
Created Date:  2011-12-14     Completed Date:  2012-04-06     Revised Date:  2012-05-11    
Medline Journal Info:
Nlm Unique ID:  9208958     Medline TA:  Hum Mol Genet     Country:  England    
Other Details:
Languages:  eng     Pagination:  26-31     Citation Subset:  IM    
Affiliation:
Howard Hughes Medical Institute and Department of Human Genetics, University of Utah, Salt Lake City, UT 84112-5331, USA.
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MeSH Terms
Descriptor/Qualifier:
Animals
Base Sequence
Disease Models, Animal*
Female
Heart / growth & development,  physiopathology
Heart Defects, Congenital / genetics*,  metabolism,  physiopathology
Homeodomain Proteins / genetics*
Humans
Male
Mice*
Mice, Inbred C57BL
Mice, Knockout
Molecular Sequence Data
Phenotype
Transcription Factors / deficiency,  genetics*
Grant Support
ID/Acronym/Agency:
NIH5R01GM021168-34/GM/NIGMS NIH HHS; R01 GM021168/GM/NIGMS NIH HHS; R37 HD030701-18/HD/NICHD NIH HHS; //Howard Hughes Medical Institute
Chemical
Reg. No./Substance:
0/Homeodomain Proteins; 0/Transcription Factors; 0/homeobox A1 protein

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine


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