Phenotypic consequences of copy number variation: insights from Smith-Magenis and Potocki-Lupski syndrome mouse models.
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Version: author
Serval ID
serval:BIB_034A965C3133
Type
Article: article from journal or magazin.
Collection
Publications
Institution
Title
Phenotypic consequences of copy number variation: insights from Smith-Magenis and Potocki-Lupski syndrome mouse models.
Journal
PLoS Biology
ISSN
1545-7885[electronic], 1544-9173[linking]
Publication state
Published
Issued date
2010
Peer-reviewed
Oui
Volume
8
Number
11
Pages
e1000543
Language
english
Abstract
A large fraction of genome variation between individuals is comprised of submicroscopic copy number variation of genomic DNA segments. We assessed the relative contribution of structural changes and gene dosage alterations on phenotypic outcomes with mouse models of Smith-Magenis and Potocki-Lupski syndromes. We phenotyped mice with 1n (Deletion/+), 2n (+/+), 3n (Duplication/+), and balanced 2n compound heterozygous (Deletion/Duplication) copies of the same region. Parallel to the observations made in humans, such variation in gene copy number was sufficient to generate phenotypic consequences: in a number of cases diametrically opposing phenotypes were associated with gain versus loss of gene content. Surprisingly, some neurobehavioral traits were not rescued by restoration of the normal gene copy number. Transcriptome profiling showed that a highly significant propensity of transcriptional changes map to the engineered interval in the five assessed tissues. A statistically significant overrepresentation of the genes mapping to the entire length of the engineered chromosome was also found in the top-ranked differentially expressed genes in the mice containing rearranged chromosomes, regardless of the nature of the rearrangement, an observation robust across different cell lineages of the central nervous system. Our data indicate that a structural change at a given position of the human genome may affect not only locus and adjacent gene expression but also "genome regulation." Furthermore, structural change can cause the same perturbation in particular pathways regardless of gene dosage. Thus, the presence of a genomic structural change, as well as gene dosage imbalance, contributes to the ultimate phenotype.
Keywords
Animals, Disease Models, Animal, Gene Dosage, Gene Expression, Mice, Phenotype, RNA, Messenger/genetics, Recombination, Genetic, Smith-Magenis Syndrome/genetics
Pubmed
Web of science
Open Access
Yes
Create date
10/03/2011 11:41
Last modification date
20/08/2019 12:25