Expanding the Genetic and Phenotypic Spectrum of Popliteal Pterygium Disorders

التفاصيل البيبلوغرافية
العنوان: Expanding the Genetic and Phenotypic Spectrum of Popliteal Pterygium Disorders
المؤلفون: Michael J. Dixon, Martine Dunnwald, Lamia Alsubaie, Jennifer Standley, Gaik Siew Ch'ng, A. Jeannette M. Hoogeboom, James O'Sullivan, Jeffrey C. Murray, Elizabeth J. Leslie, Pawina Jiramongkolchai, Ingrid M B H van der Laar, Seema Kapoor, Faroug Ababneh, Ankur Singh, Steven Goudy, J. Robert Manak, Michael L. Cunningham
المساهمون: Clinical Genetics
المصدر: American Journal of Medical Genetics Part A, 167(3), 545-552. Wiley-Liss Inc.
سنة النشر: 2015
مصطلحات موضوعية: Male, Knee Joint, Cleft Lip, DNA Mutational Analysis, Genes, Recessive, Protein Serine-Threonine Kinases, Article, Fingers, Autosomal recessive popliteal pterygium syndrome, Genetics, medicine, Humans, Exome, Knee, Eye Abnormalities, Syndactyly, Popliteal pterygium, Genetic Association Studies, Genetics (clinical), Exome sequencing, Comparative Genomic Hybridization, business.industry, Infant, Newborn, High-Throughput Nucleotide Sequencing, Infant, medicine.disease, Uniparental disomy, eye diseases, I-kappa B Kinase, Pedigree, Cleft Palate, Phenotype, Popliteal pterygium syndrome, Urogenital Abnormalities, Interferon Regulatory Factors, Mutation, Female, IRF6, business, Genome-Wide Association Study, Lower Extremity Deformities, Congenital
الوصف: The popliteal pterygia syndromes are a distinct subset of the hundreds of Mendelian orofacial clefting syndromes. Popliteal pterygia syndromes have considerable variability in severity and in the associated phenotypic features but are all characterized by cutaneous webbing across one or more major joints, cleft lip and/or palate, syndactyly, and genital malformations. Heterozygous mutations in IRF6 cause popliteal pterygium syndrome (PPS) while homozygous mutations in RIPK4 or CHUK (IKKA) cause the more severe Bartsocas-Papas syndrome (BPS) and Cocoon syndrome, respectively. In this study, we report mutations in six pedigrees with children affected with PPS or BPS. Using a combination of Sanger and exome sequencing, we report the first case of an autosomal recessive popliteal pterygium syndrome caused by homozygous mutation of IRF6 and the first case of uniparental disomy of chromosome 21 leading to a recessive disorder. We also demonstrate that mutations in RIPK4 can cause features with a range of severity along the PPS-BPS spectrum and that mutations in IKKA can cause a range of features along the BPS-Cocoon spectrum. Our findings have clinical implications for genetic counseling of families with pterygia syndromes and further implicate IRF6, RIPK4, and CHUK (IKKA) in potentially interconnected pathways governing epidermal and craniofacial development. (c) 2015 Wiley Periodicals, Inc.
تدمد: 1552-4825
DOI: 10.1002/ajmg.a.36896
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d977fcbe3d16d7b2f4a616f40139aa83
https://doi.org/10.1002/ajmg.a.36896
Rights: OPEN
رقم الانضمام: edsair.doi.dedup.....d977fcbe3d16d7b2f4a616f40139aa83
قاعدة البيانات: OpenAIRE
الوصف
تدمد:15524825
DOI:10.1002/ajmg.a.36896