Document Detail


Mutations in ribonucleic acid binding protein gene cause familial dilated cardiomyopathy.
MedLine Citation:
PMID:  19712804     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
OBJECTIVES: We sought to identify a novel gene for dilated cardiomyopathy (DCM). BACKGROUND: DCM is a heritable, genetically heterogeneous disorder that remains idiopathic in the majority of patients. Familial cases provide an opportunity to discover unsuspected molecular bases of DCM, enabling pre-clinical risk detection. METHODS: Two large families with autosomal-dominant DCM were studied. Genome-wide linkage analysis was used to identify a disease locus, followed by fine mapping and positional candidate gene sequencing. Mutation scanning was then performed in 278 unrelated subjects with idiopathic DCM, prospectively identified at the Mayo Clinic. RESULTS: Overlapping loci for DCM were independently mapped to chromosome 10q25-q26. Deoxyribonucleic acid sequencing of affected individuals in each family revealed distinct heterozygous missense mutations in exon 9 of RBM20, encoding ribonucleic acid (RNA) binding motif protein 20. Comprehensive coding sequence analyses identified missense mutations clustered within this same exon in 6 additional DCM families. Mutations segregated with DCM (peak composite logarithm of the odds score >11.49), were absent in 480 control samples, and altered residues within a highly conserved arginine/serine (RS)-rich region. Expression of RBM20 messenger RNA was confirmed in human heart tissue. CONCLUSIONS: Our findings establish RBM20 as a DCM gene and reveal a mutation hotspot in the RS domain. RBM20 is preferentially expressed in the heart and encodes motifs prototypical of spliceosome proteins that regulate alternative pre-messenger RNA splicing, thus implicating a functionally distinct gene in human cardiomyopathy. RBM20 mutations are associated with young age at diagnosis, end-stage heart failure, and high mortality.
Authors:
Katharine M Brauch; Margaret L Karst; Kathleen J Herron; Mariza de Andrade; Patricia A Pellikka; Richard J Rodeheffer; Virginia V Michels; Timothy M Olson
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Publication Detail:
Type:  Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't    
Journal Detail:
Title:  Journal of the American College of Cardiology     Volume:  54     ISSN:  1558-3597     ISO Abbreviation:  J. Am. Coll. Cardiol.     Publication Date:  2009 Sep 
Date Detail:
Created Date:  2009-08-28     Completed Date:  2009-09-23     Revised Date:  2010-09-07    
Medline Journal Info:
Nlm Unique ID:  8301365     Medline TA:  J Am Coll Cardiol     Country:  United States    
Other Details:
Languages:  eng     Pagination:  930-41     Citation Subset:  AIM; IM    
Affiliation:
Cardiovascular Genetics Laboratory, College of Medicine, Mayo Clinic, Rochester, Minnesota, USA.
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MeSH Terms
Descriptor/Qualifier:
Adolescent
Adult
Aged
Cardiomyopathy, Dilated / genetics*,  metabolism
Child, Preschool
Female
Genome-Wide Association Study
Genotype
Heterozygote
Humans
Linkage (Genetics)
Lod Score
Male
Middle Aged
Mutation, Missense*
Myocardium / metabolism
Pedigree
Phenotype
RNA, Messenger / metabolism
RNA-Binding Proteins / genetics*
Spliceosomes / genetics
Young Adult
Grant Support
ID/Acronym/Agency:
R01 HL071225/HL/NHLBI NIH HHS; R01 HL071225-06/HL/NHLBI NIH HHS
Chemical
Reg. No./Substance:
0/RNA, Messenger; 0/RNA-Binding Proteins; 0/ribonucleic acid binding motif protein 20, human
Comments/Corrections
Comment In:
J Am Coll Cardiol. 2009 Sep 1;54(10):942-3   [PMID:  19712805 ]

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


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