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Síndrome Mowat-Wilson
ORPHA:2152CID-10 · Q43.1CID-11 · LD2F.1YOMIM 235730DOENÇA RARA

A síndrome de Mowat-Wilson (MWS) é uma síndrome de anomalia congênita múltipla caracterizada por um fenótipo facial distinto, deficiência intelectual, epilepsia, doença de Hirschsprung (HSCR) e malformações congênitas variáveis.

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Introdução

O que você precisa saber de cara

📋

A síndrome de Mowat-Wilson (MWS) é uma síndrome de anomalia congênita múltipla caracterizada por um fenótipo facial distinto, deficiência intelectual, epilepsia, doença de Hirschsprung (HSCR) e malformações congênitas variáveis.

Pesquisas ativas
1 ensaio
1 total registrados no ClinicalTrials.gov
Publicações científicas
221 artigos
Último publicado: 2026 Apr 2

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
Unknown
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
0.0
Worldwide
Início
Antenatal
+ neonatal
🏥
SUS: Cobertura parcialScore: 55%
3 medicamentos CEAFCentros em: PA, PR, SC, RS, ES +8CID-10: Q43.1
🇧🇷Dados SUS / DATASUS
PROCEDIMENTOS SIGTAP (5)
0202010503
Cariótipo — bandas G, Q ou Rgenetic_test
0202010600
Pesquisa de microdeleções/microduplicações por FISHlab_test
0202010694
Sequenciamento completo do exoma (WES)rehabilitation
0202010260
Dosagem de alfa-fetoproteína
0301070040
Atendimento em reabilitação — doenças raras
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Entender a doença

Do básico ao detalhe, leia no seu ritmo

Preparando trilha educativa...

Sinais e sintomas

O que aparece no corpo e com que frequência cada sintoma acontece

Partes do corpo afetadas

🧠
Neurológico
26 sintomas
😀
Face
19 sintomas
👁️
Olhos
19 sintomas
🦴
Ossos e articulações
16 sintomas
❤️
Coração
13 sintomas
🫃
Digestivo
9 sintomas

+ 78 sintomas em outras categorias

Características mais comuns

100%prev.
Columela pendente
Frequente (79-30%)
100%prev.
Atraso no desenvolvimento da fala e da linguagem
100%prev.
Má posição dentária
Ocasional (29-5%)
100%prev.
Deficiência intelectual, grave
Frequente (79-30%)
79%prev.
Microcefalia
Frequente (79-30%)
77%prev.
Convulsão
Frequente (79-30%)
206sintomas
Muito frequente (4)
Frequente (59)
Ocasional (66)
Muito raro (18)
Sem dados (59)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 206 características clínicas mais associadas, ordenadas por frequência.

Columela pendenteLow hanging columella
Frequente (79-30%)100%
Atraso no desenvolvimento da fala e da linguagemDelayed speech and language development
Muito frequente100%
Má posição dentáriaTooth malposition
Ocasional (29-5%)100%
Deficiência intelectual, graveIntellectual disability, severe
Frequente (79-30%)100%
MicrocefaliaMicrocephaly
Frequente (79-30%)79%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico221PubMed
Últimos 10 anos132publicações
Pico201515 papers
Linha do tempo
2026Hoje · 2026📈 2015Ano de pico
Publicações por ano (últimos 10 anos)

Encontrou um erro ou informação desatualizada? Sugira uma correção →

Genética e causas

O que está alterado no DNA e como passa nas famílias

Genes associados

1 gene identificado com associação a esta condição. Padrão de herança: Autosomal dominant.

ZEB2Zinc finger E-box-binding homeobox 2Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Transcriptional inhibitor that binds to DNA sequence 5'-CACCT-3' in different promoters (PubMed:16061479, PubMed:20516212). Represses transcription of E-cadherin (PubMed:16061479). Represses expression of MEOX2 (PubMed:20516212)

LOCALIZAÇÃO

NucleusChromosome

VIAS BIOLÓGICAS (5)
Regulation of CDH11 gene transcriptionPositive Regulation of CDH1 Gene TranscriptionNegative Regulation of CDH1 Gene TranscriptionFormation of the posterior neural plateFormation of the anterior neural plate
MECANISMO DE DOENÇA

Mowat-Wilson syndrome

A complex developmental disorder characterized by intellectual disability, delayed motor development, epilepsy, microcephaly and a wide spectrum of clinically heterogeneous features suggestive of neurocristopathies at the cephalic, cardiac, and vagal levels. Affected patients show an easily recognizable facial appearance with deep set eyes and hypertelorism, medially divergent, broad eyebrows, prominent columella, pointed chin and uplifted, notched ear lobes. Some patients manifest Hirschsprung disease.

EXPRESSÃO TECIDUAL(Ubíquo)
Brain Spinal cord cervical c-1
67.9 TPM
Nervo tibial
47.6 TPM
Artéria tibial
33.1 TPM
Tecido adiposo
31.9 TPM
Substância negra
30.5 TPM
OUTRAS DOENÇAS (4)
Mowat-Wilson syndromeMowat-Wilson syndrome due to a ZEB2 point mutationlarge congenital melanocytic nevusMowat-Wilson syndrome due to monosomy 2q22
HGNC:14881UniProt:O60315

Variantes genéticas (ClinVar)

540 variantes patogênicas registradas no ClinVar.

🧬 ZEB2: NM_014795.4(ZEB2):c.3217C>T (p.His1073Tyr) ()
🧬 ZEB2: NM_014795.4(ZEB2):c.2181dup (p.Leu728fs) ()
🧬 ZEB2: NM_014795.4(ZEB2):c.278_281dup (p.Glu94fs) ()
🧬 ZEB2: NM_014795.4(ZEB2):c.74-1G>A ()
🧬 ZEB2: NM_014795.4(ZEB2):c.656G>A (p.Gly219Asp) ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 1,288 variantes classificadas pelo ClinVar.

837
451
VUS (65.0%)
Benigna (35.0%)
VARIANTES MAIS SIGNIFICATIVAS
ZEB2: NM_014795.4(ZEB2):c.1246A>G (p.Met416Val) [Uncertain significance]
ZEB2: NM_014795.4(ZEB2):c.2437G>T (p.Ala813Ser) [Uncertain significance]
ZEB2: NM_014795.4(ZEB2):c.2992A>G (p.Met998Val) [Uncertain significance]
ZEB2: NM_014795.4(ZEB2):c.418T>C (p.Cys140Arg) [Uncertain significance]
ZEB2: NM_014795.4(ZEB2):c.1319G>A (p.Gly440Asp) [Uncertain significance]

Diagnóstico

Os sinais que médicos procuram e os exames que confirmam

Carregando...

Tratamento e manejo

Remédios, cuidados de apoio e o que precisa acompanhar

Pipeline de tratamentos
Pipeline regulatório — de medicamentos já aprovados a drogas em pesquisa exploratória.
·Pré-clínico1
Medicamentos catalogadosEnsaios clínicos· 0 medicamentos · 1 ensaio
Carregando informações de tratamento...

Onde tratar no SUS

Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)

🇧🇷 Atendimento SUS — Síndrome Mowat-Wilson

Centros de Referência SUS

21 centros habilitados pelo SUS para Síndrome Mowat-Wilson

Centros para Síndrome Mowat-Wilson

Detalhes dos centros

Hospital Universitário Prof. Edgard Santos (HUPES)

R. Dr. Augusto Viana, s/n - Canela, Salvador - BA, 40110-060 · CNES 0003808

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo

Hospital de Apoio de Brasília (HAB)

AENW 3 Lote A Setor Noroeste - Plano Piloto, Brasília - DF, 70684-831 · CNES 0010456

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Estadual Infantil e Maternidade Alzir Bernardino Alves (HIABA)

Av. Min. Salgado Filho, 918 - Soteco, Vila Velha - ES, 29106-010 · CNES 6631207

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital das Clínicas da UFG

Rua 235 QD. 68 Lote Área, Nº 285, s/nº - Setor Leste Universitário, Goiânia - GO, 74605-050 · CNES 2338424

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo

Hospital das Clínicas da UFMG

Av. Prof. Alfredo Balena, 110 - Santa Efigênia, Belo Horizonte - MG, 30130-100 · CNES 2280167

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

NUPAD / Faculdade de Medicina UFMG

Av. Prof. Alfredo Balena, 189 - 5 andar - Centro, Belo Horizonte - MG, 30130-100 · CNES 2183226

Serviço de Referência

Rota
Erros Inatos do Metabolismo

Hospital Universitário João de Barros Barreto

R. dos Mundurucus, 4487 - Guamá, Belém - PA, 66073-000 · CNES 2337878

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital de Clínicas da Universidade Federal de Pernambuco

Av. Prof. Moraes Rego, 1235 - Cidade Universitária, Recife - PE, 50670-901 · CNES 2561492

Atenção Especializada

Rota
Erros Inatos do Metabolismo

Instituto de Medicina Integral Prof. Fernando Figueira (IMIP)

R. dos Coelhos, 300 - Boa Vista, Recife - PE, 50070-902 · CNES 0000647

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital de Clínicas da UFPR

R. Gen. Carneiro, 181 - Alto da Glória, Curitiba - PR, 80060-900 · CNES 2364980

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Universitário Pedro Ernesto (HUPE-UERJ)

Blvd. 28 de Setembro, 77 - Vila Isabel, Rio de Janeiro - RJ, 20551-030 · CNES 2280221

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo

Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira (IFF/Fiocruz)

Av. Rui Barbosa, 716 - Flamengo, Rio de Janeiro - RJ, 22250-020 · CNES 2269988

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Universitário Onofre Lopes (HUOL)

Av. Nilo Peçanha, 620 - Petrópolis, Natal - RN, 59012-300 · CNES 2408570

Atenção Especializada

Rota
Erros Inatos do Metabolismo

Hospital São Lucas da PUCRS

Av. Ipiranga, 6690 - Jardim Botânico, Porto Alegre - RS, 90610-000 · CNES 2232928

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo

Hospital de Clínicas de Porto Alegre (HCPA)

Rua Ramiro Barcelos, 2350 Bloco A - Av. Protásio Alves, 211 - Bloco B e C - Santa Cecília, Porto Alegre - RS, 90035-903 · CNES 2237601

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Universitário da UFSC (HU-UFSC)

R. Profa. Maria Flora Pausewang - Trindade, Florianópolis - SC, 88036-800 · CNES 2560356

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo

Hospital das Clínicas da FMUSP

R. Dr. Ovídio Pires de Campos, 225 - Cerqueira César, São Paulo - SP, 05403-010 · CNES 2077485

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital de Clínicas da UNICAMP

R. Vital Brasil, 251 - Cidade Universitária, Campinas - SP, 13083-888 · CNES 2748223

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital de Clínicas de Ribeirão Preto (HCRP-USP)

R. Ten. Catão Roxo, 3900 - Vila Monte Alegre, Ribeirão Preto - SP, 14015-010 · CNES 2082187

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Instituto da Criança e do Adolescente (ICr-HCFMUSP)

Av. Dr. Enéas Carvalho de Aguiar, 647 - Cerqueira César, São Paulo - SP, 05403-000 · CNES 2081695

Serviço de Referência

Rota
Erros Inatos do Metabolismo

UNIFESP / Hospital São Paulo

R. Napoleão de Barros, 715 - Vila Clementino, São Paulo - SP, 04024-002 · CNES 2688689

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo
Sobre os centros SUS: Estes centros são habilitados pelo Ministério da Saúde como Serviços de Referência em Doenças Raras ou Serviços de Atenção Especializada. O atendimento é pelo SUS, com encaminhamento da rede de atenção básica.

Dados de DATASUS/CNES, SBGM, ABNeuro e Ministério da Saúde. Sempre confirme a disponibilidade diretamente com o estabelecimento.

Pesquisa ativa

Ensaios clínicos abertos e novidades científicas recentes

Pesquisa e ensaios clínicos

1 ensaios clínicos encontrados, 1 ativos.

Distribuição por fase
Ver todos no ClinicalTrials.gov
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Publicações mais relevantes

Timeline de publicações
135 papers (10 anos)
#1

Transcription factor ZEB2 is essential for ureteral smooth muscle cell differentiation.

PLoS genetics2026 Jan

Mowat-Wilson Syndrome (MWS) is an autosomal dominant genetic disorder caused by heterozygous mutations or deletions in the Zinc finger E-box-binding homeobox 2 (ZEB2) gene. Congenital anomalies of the kidney and urinary tract (CAKUT), including hydroureter and hydronephrosis, have been reported in patients with MWS. However, the role of the ZEB2 gene in urinary tract development and the cellular and molecular mechanisms underlying the CAKUT phenotypes in MWS remain unknown. In this study, we examined ZEB2 expression in the developing mouse ureter and generated Zeb2 ureteral mesenchyme-specific conditional knockout mice (Zeb2 cKO) by crossing Zeb2 floxed mice with Tbx18Cre+ mice. The urinary tract of Zeb2 cKO mice and their wild-type littermates was analyzed for morphological and histological changes. Our results show that ZEB2 is expressed in TBX18+ ureteral mesenchymal cells during mouse ureter development. Deleting Zeb2 in these cells caused hydroureter and hydronephrosis, indicating obstructive uropathy. Cellular and molecular marker analysis revealed that the TAGLN+ACTA2+ ureteral smooth muscle cell (SMC) layer was absent in Zeb2 cKO mice. In contrast, the tunica adventitia cell layer was significantly expanded compared to controls. At the molecular level, Zeb2 cKO mice had significantly decreased TBX18 expression but increased SOX9 expression in the developing ureter compared to wild-type controls. Our findings demonstrate that ZEB2 is crucial for normal ureteral SMC differentiation during ureter development. Additionally, our study suggests that MWS patients may have abnormal ureteral SMC development, which contributes to the abnormalities of the urinary tract.

#2

Case Report: Identification of a de novo missense variant in the N-terminal zinc-finger domain of ZEB2 in a patient presenting with neurodevelopmental delay and recurrent pulmonary infections.

Frontiers in genetics2026

Heterozygous variants in the ZEB2 gene are known to cause Mowat-Wilson syndrome (MWS). The classical clinical spectrum of MWS includes characteristic facial features, intellectual disability, epilepsy, Hirschsprung disease (HSCR), and various congenital malformations. Reported pathogenic variants have predominantly been truncating variants or missense variants involving the C-terminal zinc-finger domain. To date, no disease-causing missense variant affecting the N-terminal zinc-finger domain has been documented. We report a 3-year-old boy presenting with characteristic facial features, global developmental delay, and recurrent respiratory tract infections. Trio-based exome sequencing identified a de novo heterozygous missense variant in ZEB2, c.652C>T (p. Arg218Trp), located within the N-terminal zinc-finger domain. The patient exhibited a phenotype distinct from classical MWS, characterized by atypical facial dysmorphisms (including an elongated face, midface hypoplasia/depression, frontal bossing, esotropia, and hypertelorism), global developmental delay, and recurrent respiratory infections. Following comprehensive rehabilitation therapy (motor, cognitive, and language training) combined with oral zinc supplementation (elemental zinc 5 mg/day, approximately 0.3 mg/kg), the patient showed a marked reduction in respiratory infections and normalization of immune parameters after 12 months of treatment. This report describes a patient with a de novo missense variant in the N-terminal zinc-finger domain of ZEB2 who presented with neurodevelopmental delay, atypical facial features, and recurrent respiratory infections, alongside a reduction in infection frequency during zinc supplementation. The variant is classified as likely pathogenic, and these observations expand the phenotypic variability potentially associated with ZEB2 variants. Additional cases and functional studies are required to confirm any causal link between the variant, the observed phenotype, and the effects of zinc supplementation.

#3

Modeling Mowat-Wilson syndrome with patient iPSCs reveals transcriptional and phenotypic defects in neural progenitors.

Neurobiology of disease2026 Jan

Zinc finger E-box-binding homeobox 2 (ZEB2) is a key transcription factor involved in multiple aspects of nervous system development, including neuronal specification, migration, and differentiation. Loss-of-function variants in ZEB2 cause Mowat-Wilson syndrome (MWS), a severe neurodevelopmental disorder characterized by intellectual disability, epilepsy, and brain structural abnormalities. In this study, we generated and characterized induced pluripotent stem cell (iPSC) lines from MWS patients carrying pathogenic ZEB2 variants. Patient-derived iPSCs retained full pluripotency and were capable of differentiating into all three germ layers, including neural lineages. Upon directed differentiation into neural progenitor cells (NPCs) and early neurons, we identified distinctive transcriptional alterations affecting neuroepithelial-to-radial glia transition and lineage specification. RNA-seq analysis revealed dysregulation of genes involved in cytoskeletal remodeling, extracellular matrix organization, and cell motility. Functional holographic live imaging confirmed a significant increase in motility behavior in MWS NPCs and early neurons, suggesting that altered cell dynamics may underlie aberrant neural circuit formation. Despite these changes, early neuronal markers such as MAP2 were expressed at comparable levels in MWS and control cells. Together, these findings uncover novel cellular and molecular phenotypes associated with ZEB2 deficiency and provide insight into how disrupted progenitor behavior and transcriptional mis-regulation may contribute to the neurodevelopmental features of MWS.

#4

Alteration of Hair Melanin in Patients With Mowat-Wilson Syndrome: The Role of the ZEB2 Gene in Regulating Melanogenesis Through SLC45A2.

Pigment cell &amp; melanoma research2025 May

Mowat-Wilson syndrome (MOWS) is a congenital disease characterized by intellectual disability, delayed motor development, characteristic facial features, epilepsy, and a wide spectrum of neurocristopathies. MOWS is caused by de novo heterozygous loss-of-function mutations or deletions in the zinc finger E-box-binding homeobox2 (ZEB2) gene, which is a multifunctional regulator of neuronal development and cancer progression/metastasis through epithelial-to-mesenchymal transition. We recognized that patients with MOWS have brown to red hair. In the present study, we report that hair from patients with MOWS has reduced eumelanin and elevated pheomelanin contents, resulting in an increased pheomelanin-to-eumelanin ratio. Furthermore, ZEB2-mutated human epidermal melanocytes show a predominance of pheomelanin biosynthesis over eumelanin and decreased expression of SLC45A2, the gene responsible for oculocutaneous albinism 4. Our results suggest that ZEB2 plays a role in mixed melanogenesis by regulating the melanosomal ion transporter gene, SLC45A2.

#5

ZEB2 Gene Pathogenic Variants Across Protein-Coding Regions and Impact on Clinical Manifestations: A Review.

International journal of molecular sciences2025 Feb 03

Mowat-Wilson syndrome (MWS) is a rare multi-system genetic disorder caused by variants in the Zinc Finger E-Box-Binding Homeobox 2 (ZEB2) gene. ZEB2 is an autosomal dominant gene containing ten exons within the canonical version transcript (Isoform: O60315-1). The ZEB2 gene encodes six functional domains and seven non-domain regions. This review provides a comprehensive summary of pathogenic variants and their associated MWS clinical characteristics, focusing on ZEB2 pathogenic variants, functional protein domains and non-domain regions with clinical features. A systematic literature search from 2001 to 2023 and of unpublished datasets found 191 individuals with reported clinical features and genotypic data. Genetic defects and clinical manifestations were examined that presumably impact on the structure and function of the ZEB2 gene, thereby causing multiple developmental defects with corresponding clinical presentation. This study found more nonsense ZEB2 variants observed within exon 8, which encodes four of the six protein domains: the CtBP-interacting domain (CID), homeodomain (HD), SMAD-binding domain (SMD or SBD) and part of the N-terminal zinc finger cluster (N-ZF), suggesting exon 8 plays a crucial role in this protein structure and function with multi-organ involvement. Exon 8 defects were found to be statistically more represented for gastrointestinal findings when compared to other exons, while frameshift defects were more often seen for the typical MWS face in non-domain protein regions. In contrast, nonsense or other types of variants in exons 3, 4 and 5 which encode only flanking non-domain regions were observed more often, compared with other exons excluding exon 8, to be specifically involved in the MWS facial gestalt, brain malformations, developmental delay and intellectual disability. Deleterious ZEB2 frameshift (45%) and nonsense (38%) gene variants were most often observed with deletions at 6% and missense at 5%. The genotype and clinical relationships in MWS can provide insights into prognosis, morbidity, clinical surveillance strategies and counseling of family members.

Publicações recentes

Ver todas no PubMed

📚 EuropePMC161 artigos no totalmostrando 129

2026

Case Report: Identification of a de novo missense variant in the N-terminal zinc-finger domain of ZEB2 in a patient presenting with neurodevelopmental delay and recurrent pulmonary infections.

Frontiers in genetics
2026

Transcription factor ZEB2 is essential for ureteral smooth muscle cell differentiation.

PLoS genetics
2025

Electro-clinical features of Mowat-Wilson syndrome: A retrospective study of 31 children in mainland China.

Epileptic disorders : international epilepsy journal with videotape
2025

B-cell deficiency caused by IKAROS deficiency in Mowat-Wilson syndrome: A pediatric case report.

Pediatrics international : official journal of the Japan Pediatric Society
2026

Modeling Mowat-Wilson syndrome with patient iPSCs reveals transcriptional and phenotypic defects in neural progenitors.

Neurobiology of disease
2025

Pyridostigmine as treatment for chronic gastrointestinal dysmotility in a child with Mowat-Wilson syndrome: A case report and literature review.

JPGN reports
2025

Impaired nutrient absorption, reduced bone mass and alterations in the gut microbiome contribute to postnatal growth retardation in a mouse model of MWS.

Scientific reports
2025

ZEB2: a multifunctional regulator of neural injury repair.

International immunopharmacology
2025

A new case of rhabdomyosarcoma in a patient with Mowat-Wilson syndrome.

Clinical dysmorphology
2025

Alteration of Hair Melanin in Patients With Mowat-Wilson Syndrome: The Role of the ZEB2 Gene in Regulating Melanogenesis Through SLC45A2.

Pigment cell &amp; melanoma research
2025

Neuropathologic Findings in Mowat-Wilson Syndrome at Autopsy, Including a Suprasellar Spindle Cell Lipoma.

Pediatric and developmental pathology : the official journal of the Society for Pediatric Pathology and the Paediatric Pathology Society
2025

Commentary - Mowat-Wilson Syndrome in Hirschsprung: Something Special or Just a Generic Problem?

Journal of pediatric surgery
2025

ZEB2 signaling is essential for ureteral smooth muscle cell differentiation and maintenance.

bioRxiv : the preprint server for biology
2025

ZEB2 Gene Pathogenic Variants Across Protein-Coding Regions and Impact on Clinical Manifestations: A Review.

International journal of molecular sciences
2025

Clinical Characteristics and Postoperative Functional Outcomes in Children With Mowat-Wilson Syndrome and Hirschsprung's Disease: A Single-center Study.

Journal of pediatric surgery
2024

[Clinical features and genetic analysis of two children with Mowat-Wilson syndrome due to variants of ZEB2 gene].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
2025

Expanding the Genetic and Phenotypic Spectrum of Mowat-Wilson Syndrome: A Study of 10 Turkish Patients With an Intrafamilial Recurrence Caused by First Intragenic Large Deletion.

American journal of medical genetics. Part A
2025

Deletions in the CDKL5 5' untranslated region lead to CDKL5 deficiency disorder.

American journal of medical genetics. Part A
2024

Mowat-Wilson syndrome: Case report.

Medicine
2024

Subtle phenotypes of Mowat-Wilson syndrome in a patient with a novel ZEB2 C-ZF domain variant.

American journal of medical genetics. Part A
2024

Human Genetics of Ventricular Septal Defect.

Advances in experimental medicine and biology
2024

Human Genetics of Atrial Septal Defect.

Advances in experimental medicine and biology
2024

Atypical Mowat-Wilson Syndrome: Dystonia, Choreoathetosis and Cognitive Features.

Movement disorders clinical practice
2024

Novel Alu insertion in the ZEB2 gene causing Mowat-Wilson syndrome.

American journal of medical genetics. Part A
2024

Mowat-Wilson Syndrome: Case Report and Review of ZEB2 Gene Variant Types, Protein Defects and Molecular Interactions.

International journal of molecular sciences
2024

Identification of the DNA methylation signature of Mowat-Wilson syndrome.

European journal of human genetics : EJHG
2024

Generation of two iPSC lines from Mowat-Wilson syndrome patients carrying heterozygous ZEB2 mutations.

Stem cell research
2024

Zeb2 drives the formation of CD11c+ atypical B cells to sustain germinal centers that control persistent infection.

Science immunology
2024

A Rare Cause of Intellectual Disability.

Cureus
2024

Unilateral progressive anterior iris adhesions in Mowat-Wilson syndrome: a new ocular finding.

Journal of AAPOS : the official publication of the American Association for Pediatric Ophthalmology and Strabismus
2023

Novel STAMBP mutations in a Chinese girl with rare symptoms of microcephaly-capillary malformation syndrome and Mowat-Wilson syndrome.

Heliyon
2024

Congenital tracheal stenosis in Mowat-Wilson syndrome with nonsense mutation of ZEB2 gene.

Pediatrics and neonatology
2023

Pathogenicity evaluation of variants of uncertain significance at exon-intron junction by splicing assay in patients with Mowat-Wilson syndrome.

European journal of medical genetics
2023

Mowat-Wilson syndrome factor ZEB2 controls early formation of human neural crest through BMP signaling modulation.

Stem cell reports
2023

"Liu-Liang-Chung" syndrome with multiple congenital anomalies and the distinctive craniofacial features caused by dominant ZEB2 gene gain mutation.

BMC pediatrics
2023

Clinical Characteristics and Novel ZEB2 Gene Mutation Analysis of Three Chinese Patients with Mowat-Wilson Syndrome.

Pharmacogenomics and personalized medicine
2023

Mutated Transcripts of ZEB2 Do Not Undergo Nonsense-Mediated Decay in Mowat-Wilson Syndrome.

Molecular syndromology
2023

Zeb2 DNA-Binding Sites in Neuroprogenitor Cells Reveal Autoregulation and Affirm Neurodevelopmental Defects, Including in Mowat-Wilson Syndrome.

Genes
2023

First Case Report of Developmental Bilateral Cataract with a Novel Mutation in the ZEB2 Gene Observed in Mowat-Wilson Syndrome.

Medicina (Kaunas, Lithuania)
2022

Physical, language, neurodevelopment and phenotype-genotype correlation of Chinese patients with Mowat-Wilson syndrome.

Frontiers in genetics
2022

ZEB2 haploinsufficient Mowat-Wilson syndrome induced pluripotent stem cells show disrupted GABAergic transcriptional regulation and function.

Frontiers in molecular neuroscience
2022

Mowat-Wilson Syndrome as a Differential Diagnosis in Patients with Congenital Heart Defects and Dysmorphic Facies.

Pharmacogenomics and personalized medicine
2022

[Clinical characteristics and genetic analysis of 3 children with Mowat-Wilson syndrome].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
2022

[Analysis of a case with Mowat-Wilson syndrome due to nonsense variant of ZEB2 gene].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
2022

A Case of Ophthalmoplegia, Hypotonia, and Developmental Delay in the Setting of Corpus Callosum Hypoplasia.

Cureus
2022

Identification of MMACHC and ZEB2 mutations causing coexistent cobalamin C disease and Mowat-Wilson syndrome in a 2-year-old girl.

Clinica chimica acta; international journal of clinical chemistry
2022

[Clinical features of epilepsy in 5 children with Mowat-Wilson syndrome].

Zhonghua er ke za zhi = Chinese journal of pediatrics
2022

Three Novel De Novo ZEB2 Variants Identified in Three Unrelated Chinese Patients With Mowat-Wilson Syndrome and A Systematic Review.

Frontiers in genetics
2022

Long-term outcome of consecutive case series of congenital isolated agenesis of corpus callosum.

Ultrasound in obstetrics &amp; gynecology : the official journal of the International Society of Ultrasound in Obstetrics and Gynecology
2022

Mowat-Wilson syndrome presenting with Shone's complex cardiac anomaly.

BMJ case reports
2022

[Analysis of ZEB2 gene variation in two patients with Mowat-Wilson syndrome].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
2021

Use of Foley's catheter as a tourniquet for the management of vascular lesion of lip in Mowat-Wilson syndrome.

Journal of the Indian Society of Pedodontics and Preventive Dentistry
2021

Further delineation and long-term evolution of electroclinical phenotype in Mowat Wilson Syndrome. A longitudinal study in 40 individuals.

Epilepsy &amp; behavior : E&amp;B
2021

A Chinese Boy with Mowat-Wilson Syndrome Caused by a 10 bp Deletion in the ZEB2 Gene.

Pharmacogenomics and personalized medicine
2021

ZEB2, the Mowat-Wilson Syndrome Transcription Factor: Confirmations, Novel Functions, and Continuing Surprises.

Genes
2021

Adhesion dynamics in the neocortex determine the start of migration and the post-migratory orientation of neurons.

Science advances
2021

Neurological Phenotype of Mowat-Wilson Syndrome.

Genes
2021

The Role of ZEB2 in Human CD8 T Lymphocytes: Clinical and Cellular Immune Profiling in Mowat-Wilson Syndrome.

International journal of molecular sciences
2021

Interstitial Deletion of 2q22.2q22.3 Involving the Entire ZEB2 Gene in a Case of Mowat-Wilson Syndrome.

Molecular syndromology
2021

A Case Report of a Prenatally Missed Mowat-Wilson Syndrome With Isolated Corpus Callosum Agenesis.

Child neurology open
2021

Clinical characteristics of Polish patients with molecularly confirmed Mowat-Wilson syndrome.

Journal of applied genetics
2021

[Clinical and genetic analysis of a patient with Mowat-Wilson syndrome].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
2020

Clinical and Molecular Spectrum of Four Patients Diagnosed with Mowat-Wilson Syndrome.

Molecular syndromology
2020

Successful treatment of drug-resistant status epilepticus in an adult patient with Mowat-Wilson syndrome: A case report.

Epilepsy &amp; behavior reports
2020

Genotype-phenotype analysis in Mowat-Wilson syndrome associated with two novel and two recurrent ZEB2 variants.

Experimental and therapeutic medicine
2021

Role of chimeric transcript formation in the pathogenesis of birth defects.

Congenital anomalies
2020

A de novo frameshift mutation in ZEB2 causes polledness, abnormal skull shape, small body stature and subfertility in Fleckvieh cattle.

Scientific reports
2020

Mowat Wilson syndrome and Hirschsprung disease: a retrospective study on functional outcomes.

Pediatric surgery international
2020

Zeb2 regulates the balance between retinal interneurons and Müller glia by inhibition of BMP-Smad signaling.

Developmental biology
2020

Targeted chromatin conformation analysis identifies novel distal neural enhancers of ZEB2 in pluripotent stem cell differentiation.

Human molecular genetics
2020

Mowat-Wilson syndrome: growth charts.

Orphanet journal of rare diseases
2020

A novel nonsense mutation of ZEB2 gene in a Chinese patient with Mowat-Wilson syndrome.

Journal of clinical laboratory analysis
2020

Submucosal Supernumerary Smooth Muscle Coat: A Common Histologic Finding in Mowat-Wilson Syndrome With or Without Hirschsprung Disease.

Pediatric and developmental pathology : the official journal of the Society for Pediatric Pathology and the Paediatric Pathology Society
2020

[Analysis of a case with Mowat-Wilson syndrome caused by ZEB2 gene variant].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
2020

Neuropathology of Mowat-Wilson Syndrome.

Pediatric and developmental pathology : the official journal of the Society for Pediatric Pathology and the Paediatric Pathology Society
2020

Mowat-Wilson syndrome in a Chinese population: A case series.

American journal of medical genetics. Part A
2020

Age-dependent epileptic encephalopathy associated with an unusual co-occurrence of ZEB2 and SCN1A variants.

Epileptic disorders : international epilepsy journal with videotape
2019

[Prenatal diagnosis of a fetus with Mowat-Wilson syndrome].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
2019

Mowat-Wilson syndrome: Generation of two human iPS cell lines (UUIGPi004A and UUIGPi005A) from siblings with a truncating ZEB2 gene variant.

Stem cell research
2019

Fetal diagnosis of Mowat-Wilson syndrome by whole exome sequencing.

American journal of medical genetics. Part A
2019

Sleep in Mowat-Wilson Syndrome: a clinical and video-polysomnographic study.

Sleep medicine
2019

[Clinical and genetic features of Mowat-Wilson syndrome: an analysis of 3 cases].

Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics
2019

[ZEB2 variation in a patient with Mowat-Wilson syndrome].

Zhonghua er ke za zhi = Chinese journal of pediatrics
2019

Functional characterization of the ZEB2 regulatory landscape.

Human molecular genetics
2019

Mowat-Wilson Syndrome Presenting With Purpura Fulminans.

Pediatrics
2019

Role of Zeb2/Sip1 in neuronal development.

Brain research
2018

Exome-first approach identified novel INDELs and gene deletions in Mowat-Wilson Syndrome patients.

Human genome variation
2018

Mutation in the Sip1 transcription factor leads to a disturbance of the preconditioning of AMPA receptors by episodes of hypoxia in neurons of the cerebral cortex due to changes in their activity and subunit composition. The protective effects of interleukin-10.

Archives of biochemistry and biophysics
2018

Incidental finding of pulmonary arterial sling during patent ductus arteriosus surgery in a patient with Mowat-Wilson syndrome.

Cardiology in the young
2019

Requirement of the Mowat-Wilson Syndrome Gene Zeb2 in the Differentiation and Maintenance of Non-photoreceptor Cell Types During Retinal Development.

Molecular neurobiology
2018

Anesthesia in Mowat-Wilson syndrome: information on 11 Italian patients.

Pediatric reports
2018

Transcriptional Regulator ZEB2 Is Essential for Bergmann Glia Development.

The Journal of neuroscience : the official journal of the Society for Neuroscience
2018

Phenotype and genotype of 87 patients with Mowat-Wilson syndrome and recommendations for care.

Genetics in medicine : official journal of the American College of Medical Genetics
2017

Mowat-Wilson syndrome presenting with fever-associated seizures.

Epileptic disorders : international epilepsy journal with videotape
2017

Syndromic Craniosynostosis Can Define New Candidate Genes for Suture Development or Result from the Non-specifc Effects of Pleiotropic Genes: Rasopathies and Chromatinopathies as Examples.

Frontiers in neuroscience
2018

Letter regarding the article "Extending the phenotype of recurrent rearrangements of 16p11.2: Deletions in mentally retarded patients without autism and in normal individuals ()" and the diagnosis of coexisting Mowat-Wilson syndrome in a patient with 16p11.2 deletion.

European journal of medical genetics
2018

Outcome of isolated agenesis of the corpus callosum: A population-based prospective study.

European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society
2017

A Novel Partial Duplication of ZEB2 and Review of ZEB2 Involvement in Mowat-Wilson Syndrome.

Molecular syndromology
2017

Electrical status epilepticus during sleep in Mowat-Wilson syndrome.

Brain &amp; development
2017

Sip-1 mutations cause disturbances in the activity of NMDA- and AMPA-, but not kainate receptors of neurons in the cerebral cortex.

Neuroscience letters
2017

Critical involvement of ZEB2 in collagen fibrillogenesis: the molecular similarity between Mowat-Wilson syndrome and Ehlers-Danlos syndrome.

Scientific reports
2017

Co-occurrence of rhabdomyosarcoma and Mowat-Wilson syndrome: is there a connection?

Clinical dysmorphology
2017

Experience of Mowat-Wilson syndrome prenatal diagnosis for a Chinese family.

Clinical case reports
2017

Incontinence and psychological symptoms in individuals with Mowat-Wilson Syndrome.

Research in developmental disabilities
2017

Neuroimaging findings in Mowat-Wilson syndrome: a study of 54 patients.

Genetics in medicine : official journal of the American College of Medical Genetics
2016

Identification of a RAI1-associated disease network through integration of exome sequencing, transcriptomics, and 3D genomics.

Genome medicine
2016

Difficult airway in Mowat-Wilson syndrome.

Journal of clinical anesthesia
2015

Pitt-Hopkins Syndrome and Differential Diagnosis: A Molecular and Clinical Challenge.

Journal of pediatric genetics
2016

Loss of Zeb2 in mesenchyme-derived nephrons causes primary glomerulocystic disease.

Kidney international
2016

Sip1 regulates the generation of the inner nuclear layer retinal cell lineages in mammals.

Development (Cambridge, England)
2016

Zeb2 recruits HDAC-NuRD to inhibit Notch and controls Schwann cell differentiation and remyelination.

Nature neuroscience
2016

Anaesthetic management of Mowat-Wilson syndrome.

Indian journal of anaesthesia
2016

Novel Zeb2 gene variation in the Mowat Wilson syndrome (MWS).

Journal of pediatric surgery
2016

A Diagnosis to Consider in Intellectual Disability: Mowat-Wilson Syndrome.

Journal of child neurology
2016

Early Infantile Epileptic Encephalopathy with a de novo variant in ZEB2 identified by exome sequencing.

European journal of medical genetics
2015

A de novo triplication on 2q22.3 including the entire ZEB2 gene associated with global developmental delay, multiple congenital anomalies and behavioral abnormalities.

Molecular cytogenetics
2016

Sleep disturbance in Mowat-Wilson syndrome.

American journal of medical genetics. Part A
2016

A new risk locus in the ZEB2 gene for schizophrenia in the Han Chinese population.

Progress in neuro-psychopharmacology &amp; biological psychiatry
2015

Psychopharmacological Management of Problem Behaviors in Mowat-Wilson Syndrome.

Journal of child and adolescent psychopharmacology
2015

De novo inbred heterozygous Zeb2/Sip1 mutant mice uniquely generated by germ-line conditional knockout exhibit craniofacial, callosal and behavioral defects associated with Mowat-Wilson syndrome.

Human molecular genetics
2015

Zeb2: A multifunctional regulator of nervous system development.

Progress in neurobiology
2015

Hirschsprung's disease in children with Mowat-Wilson syndrome.

Pediatric surgery international
2015

"CHARGE-like presentation, craniosynostosis and mild Mowat-Wilson Syndrome diagnosed by recognition of the distinctive facial gestalt in a cohort of 28 new cases" American Journal of Medical Genetics Part A. 164:2557-2566, 2014.

American journal of medical genetics. Part A
2015

Polymicrogyria in a 10-month-old boy with Mowat-Wilson syndrome.

American journal of medical genetics. Part A
2015

Clinical spectrum of eye malformations in four patients with Mowat-Wilson syndrome.

American journal of medical genetics. Part A
2015

Hirschsprung disease associated with Mowat-Wilson syndrome: report of a case.

Nutricion hospitalaria
2015

Sip1 downstream Effector ninein controls neocortical axonal growth, ipsilateral branching, and microtubule growth and stability.

Neuron
2015

Mowat-Wilson syndrome: neurological and molecular study in seven patients.

Arquivos de neuro-psiquiatria
2015

Exploring the genetic counselor's role in facilitating meaning-making: rare disease diagnoses.

Journal of genetic counseling
Ver todos os 161 no EuropePMC

Associações

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Comunidades

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Doenças relacionadas

Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico

Ordenadas pelo número de sintomas em comum.

Referências e fontes

Bases de dados externas citadas neste artigo

Publicações científicas

Artigos indexados no PubMed ligados a esta doença no grafo RarasNet — título, periódico e PMID direto da fonte, sem intermediação de IA.

  1. Transcription factor ZEB2 is essential for ureteral smooth muscle cell differentiation.
    PLoS genetics· 2026· PMID 41576029mais citado
  2. Case Report: Identification of a de novo missense variant in the N-terminal zinc-finger domain of ZEB2 in a patient presenting with neurodevelopmental delay and recurrent pulmonary infections.
    Frontiers in genetics· 2026· PMID 41788411mais citado
  3. Modeling Mowat-Wilson syndrome with patient iPSCs reveals transcriptional and phenotypic defects in neural progenitors.
    Neurobiology of disease· 2026· PMID 41325909mais citado
  4. Alteration of Hair Melanin in Patients With Mowat-Wilson Syndrome: The Role of the ZEB2 Gene in Regulating Melanogenesis Through SLC45A2.
    Pigment cell &amp; melanoma research· 2025· PMID 40415661mais citado
  5. ZEB2 Gene Pathogenic Variants Across Protein-Coding Regions and Impact on Clinical Manifestations: A Review.
    International journal of molecular sciences· 2025· PMID 39941075mais citado
  6. Profiles and Predictors of Family Functioning in Families of Children with Mowat-Wilson Syndrome: A Cross-Sectional Survey.
    Healthcare (Basel)· 2026· PMID 41975932recente
  7. Whole Genome Sequencing Reveals a RET Enhancer Risk Haplotype Associated with Hirschsprung Disease in Mowat Wilson Syndrome.
    medRxiv· 2026· PMID 41929340recente
  8. Epilepsy in Chinese Children With Mowat-Wilson Syndrome: Two Case Reports and Literature Review.
    J Paediatr Child Health· 2026· PMID 41896167recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:2152(Orphanet)
  2. OMIM OMIM:235730(OMIM)
  3. MONDO:0009341(MONDO)
  4. GARD:9673(GARD (NIH))
  5. Variantes catalogadas(ClinVar)
  6. Busca completa no PubMed(PubMed)
  7. Q2757585(Wikidata)

Dados compilados pelo RarasNet a partir de fontes abertas (Orphanet, OMIM, MONDO, PubMed/EuropePMC, ClinicalTrials.gov, DATASUS, PCDT/MS). Este conteúdo é informativo e não substitui avaliação médica.

Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar

Síndrome Mowat-Wilson
Compêndio · Raras BR

Síndrome Mowat-Wilson

ORPHA:2152 · MONDO:0009341
🇧🇷 Brasil SUS
CEAF
1BD-Penicilamina1ATrientinaAcetato de zinco
Geral
Prevalência
Unknown
Herança
Autosomal dominant
CID-10
Q43.1 · Doença de Hirschsprung
CID-11
Ensaios
1 ativos
Início
Antenatal, Neonatal
Prevalência
0.0 (Worldwide)
MedGen
UMLS
C1856113
EuropePMC
Wikidata
Papers 10a
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