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European Journal of Medical Genetics
Article . 2014 . Peer-reviewed
License: Elsevier TDM
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Severe ipsilateral musculoskeletal involvement in a Cornelia de Lange patient with a novel NIPBL mutation

Authors: Baquero Montoya, Carolina; Gil Rodríguez, María Concepción; Hernández Marcos, María; Teresa Rodrigo, María Esperanza; Vicente Gabas, Alicia; Bernal, María Luisa; Casale, Cesar Horacio; +11 Authors

Severe ipsilateral musculoskeletal involvement in a Cornelia de Lange patient with a novel NIPBL mutation

Abstract

Cornelia de Lange Syndrome (CdLS) is a congenital autosomal dominant (NIPBL, SMC3 and RAD21) or X-linked (SMC1A and HDAC8) disorder characterized by facial dysmorphism, pre and postnatal growth retardation, developmental delay and/or intellectual disability, and multiorgan involvement. Musculoskeletal malformations are usually bilateral and affect mainly the upper limbs; the range goes from brachyclinodactyly to severe reduction defects. Instead lower extremities are usually less and mildly involved. Here, we report on a 3-year-old Senegalese boy with typical craniofacial CdLS features, pre and postnatal growth retardation, atrial septal defect, developmental delay and right ipsilateral limb malformations, consistent with oligodactyly of the 3rd and 4th fingers, tibial agenesis and fibula hypoplasia. Exome sequencing and Sanger sequencing showed a novel missense mutation in NIPBL gene (c.6647A>G; p.(Tyr2216Cys)), which affects a conserved residue located within NIPBL HEAT repeat elements. Pyrosequencing analysis of NIPBL gene, disclosed similar levels of wild-type and mutated alleles in DNA and RNA samples from all tissues analyzed (oral mucosa epithelial cells, peripheral blood leukocytes and fibroblasts). These findings indicated the absence of somatic mosaicism, despite of the segmental asymmetry of the limbs, and confirmed biallelic expression for NIPBL transcripts, respectively. Additionally, conditions like Split-hand/foot malformation with long-bone deficiency secondary to duplication of BHLHA9 gene have been ruled out by the array-CGH and MLPA analysis. To our knowledge, this is the first CdLS patient described with major ipsilateral malformations of both the upper and lower extremities, that even though this finding could be due to a random event, expands the spectrum of limb reduction defects in CdLS.

Country
Argentina
Keywords

CDLS, Exome sequencing, Male, Models, Molecular, NIPBL mutation, CORNELIA DE LANGE SYNDROME, Protein Conformation, Molecular Sequence Data, HEAT repeat, Cell Cycle Proteins, EXOME SEQUENCING, https://purl.org/becyt/ford/1.6, De Lange Syndrome, Gene Order, Cornelia de Lange Syndrome, Humans, Exome, IPSILATERAL, Amino Acid Sequence, https://purl.org/becyt/ford/1, Alleles, HEAT REPEAT, CdLS, Comparative Genomic Hybridization, BHLHA9 duplication, NIPBL MUTATION, High-Throughput Nucleotide Sequencing, Infant, Proteins, Musculoskeletal Abnormalities, Pedigree, Ipsilateral, Phenotype, BHLHA9 DUPLICATION, Mutation, Musculoskeletal involvement, Sequence Alignment, MUSCULOSKELETAL INVOLVEMENT

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selected citations
These citations are derived from selected sources.
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
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