Deep Phenotyping and Genetic Characterization of a Cohort of 70 Individuals With 5p Minus Syndrome

Chromosome-5p minus syndrome (5p-Sd, OMIM #123450) formerly known as Cri duChat syndrome results from the loss of genetic material at the distal region of the short arm of chromosome 5. It is a neurodevelopmental disorder of genetic cause. So far, about 400 patients have been reported worldwide. Ind...

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Detalhes bibliográficos
Autores: Nevado, Julian, Bel Fenellos, María Cristina, Sandoval-Talamantes, Ana Karen, Hernández Estrada, Adolfo, Biencinto López, Chantal-María, Martínez-Fernández, María Luisa, Barrúz, Pilar, Santos-Simarro, Fernando, Mori-Álvarez, María Ángeles, Mansilla, Elena, García-Santiago, Fé Amalia, Valcorba, Isabel, Sáenz-Rico de Santiago, María Belén, Martínez-Frías, María Luisa, Lapunzina, Pablo
Formato: artículo
Fecha de publicación:2021
País:España
Recursos:Universidad Complutense de Madrid (UCM)
Repositorio:Docta Complutense
Idioma:español
OAI Identifier:oai:docta.ucm.es:20.500.14352/100143
Acesso em linha:https://hdl.handle.net/20.500.14352/100143
Access Level:acceso abierto
Palavra-chave:37.013
37.012
616-053.2
159.922
612.65-053.2
5p-minus syndrome
Intellectual disabilities
Cri du chat
Subtelomeric deletion
Behavior problems
Ciencias Biomédicas
Ciencias Sociales
Métodos de investigación en educación
61 Psicología
58 Pedagogía
32 Ciencias Médicas
Descrição
Resumo:Chromosome-5p minus syndrome (5p-Sd, OMIM #123450) formerly known as Cri duChat syndrome results from the loss of genetic material at the distal region of the short arm of chromosome 5. It is a neurodevelopmental disorder of genetic cause. So far, about 400 patients have been reported worldwide. Individuals affected by this syndrome have large phenotypic heterogeneity. However, a specific phenotype has emerged including global developmental delay, microcephaly, delayed speech, some dysmorphic features, and a characteristic and monochromatic high-pitch voice, resembling a cat’s cry. We here describe a cohort of 70 patients with clinical features of 5p- Sd characterized by means of deep phenotyping, SNP arrays, and other genetic approaches. Individuals have a great clinical and molecular heterogeneity, which can be partially explained by the existence of additional significant genomic rearrangements in around 39% of cases. Thus, our data showed significant statistical differences between subpopulations (simple 5p deletions versus 5p deletions plus additional rearrangements) of the cohort. We also determined significant “functional” differences between male andfemale individuals