Raras
Buscar doenças, sintomas, genes...
Síndrome Coffin-Siris
ORPHA:1465CID-10 · Q87.1CID-11 · LD27.0YDOENÇA RARA

A síndrome de Coffin-Siris (SCS) é uma condição genética rara e de nascença que afeta diversas partes do corpo. Ela se caracteriza pela falta ou desenvolvimento incompleto do osso da ponta ou da unha do quinto dedo, atraso no desenvolvimento, deficiência intelectual, traços faciais marcantes e outras manifestações clínicas que podem variar.

Mantido por Agente Raras·Colaborar como especialista →

Introdução

O que você precisa saber de cara

📋

A síndrome de Coffin-Siris (SCS) é uma condição genética rara e de nascença que afeta diversas partes do corpo. Ela se caracteriza pela falta ou desenvolvimento incompleto do osso da ponta ou da unha do quinto dedo, atraso no desenvolvimento, deficiência intelectual, traços faciais marcantes e outras manifestações clínicas que podem variar.

Publicações científicas
351 artigos
Último publicado: 2026

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
<1 / 1 000 000
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
0.0
Worldwide
Casos conhecidos
190
pacientes catalogados
Início
Antenatal
+ neonatal
🏥
SUS: Cobertura mínimaScore: 15%
CID-10: Q87.1
🇧🇷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
Você se identifica com essa condição?
O Raras está aqui pra te apoiar — com ou sem diagnóstico

Encontrou um erro ou informação desatualizada? Sugira uma correção →

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

😀
Face
37 sintomas
🦴
Ossos e articulações
33 sintomas
🧠
Neurológico
25 sintomas
🫃
Digestivo
13 sintomas
📏
Crescimento
12 sintomas
🧬
Pele e cabelo
10 sintomas

+ 90 sintomas em outras categorias

Características mais comuns

90%prev.
Dificuldades alimentares
Muito frequente (99-80%)
90%prev.
Hipertricose
Muito frequente (99-80%)
90%prev.
Vermelhão do lábio inferior espesso
Muito frequente (99-80%)
90%prev.
Sobrancelha espessa
Muito frequente (99-80%)
90%prev.
Traços faciais grosseiros
Muito frequente (99-80%)
90%prev.
Boca larga
Muito frequente (99-80%)
260sintomas
Muito frequente (8)
Frequente (37)
Ocasional (16)
Muito raro (4)
Sem dados (195)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 260 características clínicas mais associadas, ordenadas por frequência.

Dificuldades alimentaresFeeding difficulties
Muito frequente (99-80%)90%
HipertricoseHypertrichosis
Muito frequente (99-80%)90%
Vermelhão do lábio inferior espessoThick lower lip vermilion
Muito frequente (99-80%)90%
Sobrancelha espessaThick eyebrow
Muito frequente (99-80%)90%
Traços faciais grosseirosCoarse facial features
Muito frequente (99-80%)90%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico351PubMed
Últimos 10 anos200publicações
Pico202236 papers
Linha do tempo
2026Hoje · 2026📈 2022Ano 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

12 genes identificados com associação a esta condição. Padrão de herança: Autosomal dominant.

ARID1AAT-rich interactive domain-containing protein 1ADisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Involved in transcriptional activation and repression of select genes by chromatin remodeling (alteration of DNA-nucleosome topology). Component of SWI/SNF chromatin remodeling complexes that carry out key enzymatic activities, changing chromatin structure by altering DNA-histone contacts within a nucleosome in an ATP-dependent manner. Binds DNA non-specifically. Belongs to the neural progenitors-specific chromatin remodeling complex (npBAF complex) and the neuron-specific chromatin remodeling c

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (8)
Positive Regulation of CDH1 Gene TranscriptionRUNX1 interacts with co-factors whose precise effect on RUNX1 targets is not knownRMTs methylate histone argininesFormation of neuronal progenitor and neuronal BAF (npBAF and nBAF)Formation of the canonical BAF (cBAF) complex
MECANISMO DE DOENÇA

Coffin-Siris syndrome 2

A form of Coffin-Siris syndrome, a congenital multiple malformation syndrome with broad phenotypic and genetic variability. Cardinal features are intellectual disability, coarse facial features, hypertrichosis, and hypoplastic or absent fifth digit nails or phalanges. Additional features include malformations of the cardiac, gastrointestinal, genitourinary, and/or central nervous systems. Sucking/feeding difficulties, poor growth, ophthalmologic abnormalities, hearing impairment, and spinal anomalies are common findings. Both autosomal dominant and autosomal recessive inheritance patterns have been reported.

OUTRAS DOENÇAS (2)
intellectual disability, autosomal dominant 14Coffin-Siris syndrome
HGNC:11110UniProt:O14497
BICRABRD4-interacting chromatin-remodeling complex-associated proteinDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Component of SWI/SNF chromatin remodeling subcomplex GBAF that carries out key enzymatic activities, changing chromatin structure by altering DNA-histone contacts within a nucleosome in an ATP-dependent manner (PubMed:29374058). May play a role in BRD4-mediated gene transcription (PubMed:21555454)

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (1)
Formation of the non-canonical BAF (ncBAF) complex
MECANISMO DE DOENÇA

Coffin-Siris syndrome 12

A form of Coffin-Siris syndrome, a congenital multiple malformation syndrome with broad phenotypic and genetic variability. Cardinal features are intellectual disability, coarse facial features, hypertrichosis, and hypoplastic or absent fifth digit nails or phalanges. Additional features include malformations of the cardiac, gastrointestinal, genitourinary, and/or central nervous systems. Sucking/feeding difficulties, poor growth, ophthalmologic abnormalities, hearing impairment, and spinal anomalies are common findings. CSS12 is an autosomal dominant form characterized by global developmental delay with variably impaired intellectual development, speech and language delay, and behavioral abnormalities, such as autism or hyperactivity. Most CSS12 patients do not have the classic hypoplastic fifth digit/nail abnormalities that are often observed in other forms the disease.

OUTRAS DOENÇAS (1)
Coffin-Siris syndrome 12
HGNC:HGNC:4332UniProt:Q9NZM4
ARID1BAT-rich interactive domain-containing protein 1BDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Involved in transcriptional activation and repression of select genes by chromatin remodeling (alteration of DNA-nucleosome topology). Component of SWI/SNF chromatin remodeling complexes that carry out key enzymatic activities, changing chromatin structure by altering DNA-histone contacts within a nucleosome in an ATP-dependent manner. Belongs to the neural progenitors-specific chromatin remodeling complex (npBAF complex) and the neuron-specific chromatin remodeling complex (nBAF complex). Durin

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (6)
RUNX1 interacts with co-factors whose precise effect on RUNX1 targets is not knownRMTs methylate histone argininesFormation of neuronal progenitor and neuronal BAF (npBAF and nBAF)Formation of the canonical BAF (cBAF) complexRegulation of MITF-M-dependent genes involved in pigmentation
MECANISMO DE DOENÇA

Coffin-Siris syndrome 1

A form of Coffin-Siris syndrome, a congenital multiple malformation syndrome with broad phenotypic and genetic variability. Cardinal features are intellectual disability, coarse facial features, hypertrichosis, and hypoplastic or absent fifth digit nails or phalanges. Additional features include malformations of the cardiac, gastrointestinal, genitourinary, and/or central nervous systems. Sucking/feeding difficulties, poor growth, ophthalmologic abnormalities, hearing impairment, and spinal anomalies are common findings. Both autosomal dominant and autosomal recessive inheritance patterns have been reported.

OUTRAS DOENÇAS (3)
Coffin-Siris syndrome 1chromosome 6q24-q25 deletion syndromeCoffin-Siris syndrome
HGNC:18040UniProt:Q8NFD5
SMARCE1SWI/SNF-related matrix-associated actin-dependent regulator of chromatin subfamily E member 1Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Involved in transcriptional activation and repression of select genes by chromatin remodeling (alteration of DNA-nucleosome topology). Component of SWI/SNF chromatin remodeling complexes that carry out key enzymatic activities, changing chromatin structure by altering DNA-histone contacts within a nucleosome in an ATP-dependent manner. Belongs to the neural progenitors-specific chromatin remodeling complex (npBAF complex) and the neuron-specific chromatin remodeling complex (nBAF complex). Durin

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (8)
RUNX1 interacts with co-factors whose precise effect on RUNX1 targets is not knownRMTs methylate histone argininesFormation of neuronal progenitor and neuronal BAF (npBAF and nBAF)Formation of the embryonic stem cell BAF (esBAF) complexFormation of the polybromo-BAF (pBAF) complex
MECANISMO DE DOENÇA

Meningioma

A common neoplasm of the central nervous system derived from arachnoidal cells. The majority of meningiomas are well differentiated vascular tumors which grow slowly and have a low potential to be invasive, although malignant subtypes occur. Most cases are sporadic. Familial occurrence of meningioma is rare.

EXPRESSÃO TECIDUAL(Ubíquo)
Útero
58.4 TPM
Cervix Endocervix
51.0 TPM
Fibroblastos
47.8 TPM
Artéria tibial
45.6 TPM
Cervix Ectocervix
45.5 TPM
OUTRAS DOENÇAS (5)
Coffin-Siris syndrome 5familial multiple meningiomameningiomaCoffin-Siris syndrome
HGNC:11109UniProt:Q969G3
SOX11Transcription factor SOX-11Disease-causing germline mutation(s) inRestrito
FUNÇÃO

Transcription factor that acts as a transcriptional activator (PubMed:24886874, PubMed:26543203). Binds cooperatively with POU3F2/BRN2 or POU3F1/OCT6 to gene promoters, which enhances transcriptional activation (By similarity). Acts as a transcriptional activator of TEAD2 by binding to its gene promoter and first intron (By similarity). Plays a redundant role with SOX4 and SOX12 in cell survival of developing tissues such as the neural tube, branchial arches and somites, thereby contributing to

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (1)
Regulation of MITF-M-dependent genes involved in apoptosis
MECANISMO DE DOENÇA

Intellectual developmental disorder with microcephaly and with or without ocular malformations or hypogonadotropic hypogonadism

An autosomal dominant disorder characterized by developmental delay, impaired intellectual development and microcephaly. Affected individuals may also have oculomotor apraxia, ocular malformations including coloboma, lens abnormalities and microphthalmia, and hypogonadotropic hypogonadism. Some patients may have finger clinodactyly and hypoplastic distal phalanges with nail hypoplasia, especially of the fifth digits.

EXPRESSÃO TECIDUAL(Baixa expressão)
Hipotálamo
2.3 TPM
Brain Nucleus accumbens basal ganglia
2.3 TPM
Cólon sigmoide
2.0 TPM
Brain Caudate basal ganglia
1.5 TPM
Brain Spinal cord cervical c-1
1.4 TPM
OUTRAS DOENÇAS (2)
intellectual developmental disorder with microcephaly and with or without ocular malformations or hypogonadotropic hypogonadismCoffin-Siris syndrome
HGNC:11191UniProt:P35716
SMARCC2SWI/SNF complex subunit SMARCC2Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Involved in transcriptional activation and repression of select genes by chromatin remodeling (alteration of DNA-nucleosome topology). Component of SWI/SNF chromatin remodeling complexes that carry out key enzymatic activities, changing chromatin structure by altering DNA-histone contacts within a nucleosome in an ATP-dependent manner (PubMed:11018012). Can stimulate the ATPase activity of the catalytic subunit of these complexes (PubMed:10078207). May be required for CoREST dependent repression

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (8)
RUNX1 interacts with co-factors whose precise effect on RUNX1 targets is not knownRMTs methylate histone argininesFormation of neuronal progenitor and neuronal BAF (npBAF and nBAF)Formation of the embryonic stem cell BAF (esBAF) complexFormation of the polybromo-BAF (pBAF) complex
MECANISMO DE DOENÇA

Coffin-Siris syndrome 8

A form of Coffin-Siris syndrome, a congenital multiple malformation syndrome with broad phenotypic and genetic variability. Cardinal features are intellectual disability, coarse facial features, hypertrichosis, and hypoplastic or absent fifth digit nails or phalanges. Additional features include malformations of the cardiac, gastrointestinal, genitourinary, and/or central nervous systems. Sucking/feeding difficulties, poor growth, ophthalmologic abnormalities, hearing impairment, and spinal anomalies are common findings. CSS8 patients manifest prominent speech impairment, hypotonia, feeding difficulties, behavioral abnormalities, and dysmorphic features such as hypertrichosis, thick eyebrows, thin upper lip vermilion, and upturned nose. CSS8 inheritance is autosomal dominant.

EXPRESSÃO TECIDUAL(Ubíquo)
Cérebro - Hemisfério cerebelar
137.3 TPM
Cerebelo
136.4 TPM
Tireoide
130.7 TPM
Ovário
127.6 TPM
Cervix Endocervix
125.2 TPM
OUTRAS DOENÇAS (2)
Coffin-Siris syndrome 8Coffin-Siris syndrome
HGNC:11105UniProt:Q8TAQ2
SOX4Transcription factor SOX-4Disease-causing germline mutation(s) inDesconhecido
FUNÇÃO

Transcriptional activator that binds with high affinity to the T-cell enhancer motif 5'-AACAAAG-3' motif (PubMed:30661772). Required for IL17A-producing Vgamma2-positive gamma-delta T-cell maturation and development, via binding to regulator loci of RORC to modulate expression (By similarity). Involved in skeletal myoblast differentiation by promoting gene expression of CALD1 (PubMed:26291311)

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (1)
Deactivation of the beta-catenin transactivating complex
MECANISMO DE DOENÇA

Intellectual developmental disorder with speech delay and dysmorphic facies

An autosomal dominant disorder characterized by mild to severe intellectual disability, global developmental delay, mild but distinct facial dysmorphism, fifth finger clinodactyly, and small stature. Hypotonia, ventricular septal defect, and spastic quadriparesis may also be present.

EXPRESSÃO TECIDUAL(Ubíquo)
Ovário
65.6 TPM
Cervix Endocervix
44.3 TPM
Útero
30.1 TPM
Rim - Medula
28.8 TPM
Cervix Ectocervix
26.8 TPM
OUTRAS DOENÇAS (2)
Coffin-Siris syndrome 10Coffin-Siris syndrome
HGNC:11200UniProt:Q06945
DPF2Zinc finger protein ubi-d4Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Plays an active role in transcriptional regulation by binding modified histones H3 and H4 (PubMed:27775714, PubMed:28533407). Is a negative regulator of myeloid differentiation of hematopoietic progenitor cells (PubMed:28533407). Might also have a role in the development and maturation of lymphoid cells (By similarity). Involved in the regulation of non-canonical NF-kappa-B pathway (PubMed:20460684)

LOCALIZAÇÃO

NucleusCytoplasm

VIAS BIOLÓGICAS (5)
Formation of neuronal progenitor and neuronal BAF (npBAF and nBAF)Formation of the embryonic stem cell BAF (esBAF) complexFormation of the canonical BAF (cBAF) complexRegulation of MITF-M-dependent genes involved in pigmentationRegulation of endogenous retroelements by Piwi-interacting RNAs (piRNAs)
MECANISMO DE DOENÇA

Coffin-Siris syndrome 7

A form of Coffin-Siris syndrome, a congenital multiple malformation syndrome with broad phenotypic and genetic variability. Cardinal features are intellectual disability, coarse facial features, hypertrichosis, and hypoplastic or absent fifth digit nails or phalanges. Additional features include malformations of the cardiac, gastrointestinal, genitourinary, and/or central nervous systems. Sucking/feeding difficulties, poor growth, ophthalmologic abnormalities, hearing impairment, and spinal anomalies are common findings. Both autosomal dominant and autosomal recessive inheritance patterns have been reported. CSS7 inheritance is autosomal dominant.

EXPRESSÃO TECIDUAL(Ubíquo)
Útero
88.8 TPM
Cervix Endocervix
83.4 TPM
Testículo
83.3 TPM
Ovário
82.0 TPM
Fallopian Tube
71.8 TPM
OUTRAS DOENÇAS (2)
Coffin-Siris syndrome 7Coffin-Siris syndrome
HGNC:9964UniProt:Q92785
SMARCB1SWI/SNF-related matrix-associated actin-dependent regulator of chromatin subfamily B member 1Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Core component of the BAF (hSWI/SNF) complex. This ATP-dependent chromatin-remodeling complex plays important roles in cell proliferation and differentiation, in cellular antiviral activities and inhibition of tumor formation. The BAF complex is able to create a stable, altered form of chromatin that constrains fewer negative supercoils than normal. This change in supercoiling would be due to the conversion of up to one-half of the nucleosomes on polynucleosomal arrays into asymmetric structures

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (8)
RUNX1 interacts with co-factors whose precise effect on RUNX1 targets is not knownRMTs methylate histone argininesFormation of neuronal progenitor and neuronal BAF (npBAF and nBAF)Formation of the embryonic stem cell BAF (esBAF) complexFormation of the polybromo-BAF (pBAF) complex
MECANISMO DE DOENÇA

Rhabdoid tumor predisposition syndrome 1

A familial cancer syndrome predisposing to renal or extrarenal malignant rhabdoid tumors and to a variety of tumors of the central nervous system, including choroid plexus carcinoma, medulloblastoma, and central primitive neuroectodermal tumors. Rhabdoid tumors are the most aggressive and lethal malignancies occurring in early childhood.

EXPRESSÃO TECIDUAL(Ubíquo)
Linfócitos
136.4 TPM
Testículo
110.5 TPM
Cérebro - Hemisfério cerebelar
98.7 TPM
Cerebelo
93.5 TPM
Ovário
93.2 TPM
OUTRAS DOENÇAS (9)
rhabdoid tumor predisposition syndrome 1intellectual disability, autosomal dominant 15familial multiple meningiomaschwannomatosis
HGNC:11103UniProt:Q12824
SMARCA4SWI/SNF-related matrix-associated actin-dependent regulator of chromatin subfamily A member 4Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

ATPase involved in transcriptional activation and repression of select genes by chromatin remodeling (alteration of DNA-nucleosome topology). Component of SWI/SNF chromatin remodeling complexes that carry out key enzymatic activities, changing chromatin structure by altering DNA-histone contacts within a nucleosome in an ATP-dependent manner (PubMed:15075294, PubMed:29374058, PubMed:30339381, PubMed:32459350). Component of the CREST-BRG1 complex, a multiprotein complex that regulates promoter ac

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (10)
Formation of the beta-catenin:TCF transactivating complexNegative Regulation of CDH1 Gene TranscriptionRUNX1 interacts with co-factors whose precise effect on RUNX1 targets is not knownRMTs methylate histone argininesFormation of neuronal progenitor and neuronal BAF (npBAF and nBAF)
MECANISMO DE DOENÇA

Rhabdoid tumor predisposition syndrome 2

A familial cancer syndrome predisposing to renal or extrarenal malignant rhabdoid tumors and to a variety of tumors of the central nervous system, including choroid plexus carcinoma, medulloblastoma, and central primitive neuroectodermal tumors. Rhabdoid tumors are the most aggressive and lethal malignancies occurring in early childhood.

EXPRESSÃO TECIDUAL(Ubíquo)
Testículo
81.7 TPM
Cérebro - Hemisfério cerebelar
65.7 TPM
Cerebelo
62.6 TPM
Linfócitos
61.0 TPM
Esôfago - Mucosa
57.8 TPM
OUTRAS DOENÇAS (7)
intellectual disability, autosomal dominant 16otosclerosis 12Coffin-Siris syndromeovarian small cell carcinoma
HGNC:11100UniProt:P51532
ARID2AT-rich interactive domain-containing protein 2Disease-causing germline mutation(s) (loss of function) inAltamente restrito
FUNÇÃO

Involved in transcriptional activation and repression of select genes by chromatin remodeling (alteration of DNA-nucleosome topology). Required for the stability of the SWI/SNF chromatin remodeling complex SWI/SNF-B (PBAF). May be involved in targeting the complex to different genes. May be involved in regulating transcriptional activation of cardiac genes

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (3)
RUNX1 interacts with co-factors whose precise effect on RUNX1 targets is not knownRMTs methylate histone argininesFormation of the polybromo-BAF (pBAF) complex
MECANISMO DE DOENÇA

Coffin-Siris syndrome 6

A form of Coffin-Siris syndrome, a congenital multiple malformation syndrome with broad phenotypic and genetic variability. Cardinal features are intellectual disability, coarse facial features, hypertrichosis, and hypoplastic or absent fifth digit nails or phalanges. Additional features include malformations of the cardiac, gastrointestinal, genitourinary, and/or central nervous systems. Sucking/feeding difficulties, poor growth, ophthalmologic abnormalities, hearing impairment, and spinal anomalies are common findings. Both autosomal dominant and autosomal recessive inheritance patterns have been reported. CSS6 inheritance is autosomal dominant.

OUTRAS DOENÇAS (2)
Coffin-Siris syndrome 6Coffin-Siris syndrome
HGNC:18037UniProt:Q68CP9
SMARCD1SWI/SNF-related matrix-associated actin-dependent regulator of chromatin subfamily D member 1Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Involved in transcriptional activation and repression of select genes by chromatin remodeling (alteration of DNA-nucleosome topology). Component of SWI/SNF chromatin remodeling complexes that carry out key enzymatic activities, changing chromatin structure by altering DNA-histone contacts within a nucleosome in an ATP-dependent manner (PubMed:29374058, PubMed:8804307). Belongs to the neural progenitors-specific chromatin remodeling complex (npBAF complex) and the neuron-specific chromatin remode

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (9)
RUNX1 interacts with co-factors whose precise effect on RUNX1 targets is not knownRMTs methylate histone argininesFormation of neuronal progenitor and neuronal BAF (npBAF and nBAF)Formation of the embryonic stem cell BAF (esBAF) complexFormation of the polybromo-BAF (pBAF) complex
MECANISMO DE DOENÇA

Coffin-Siris syndrome 11

A form of Coffin-Siris syndrome, a congenital multiple malformation syndrome with broad phenotypic and genetic variability. Cardinal features are intellectual disability, coarse facial features, hypertrichosis, and hypoplastic or absent fifth digit nails or phalanges. Additional features include malformations of the cardiac, gastrointestinal, genitourinary, and/or central nervous systems. Sucking/feeding difficulties, poor growth, ophthalmologic abnormalities, hearing impairment, and spinal anomalies are common findings. CSS11 is an autosomal dominant form characterized by developmental delay, intellectual disability, hypotonia, feeding difficulties, and small hands and feet.

EXPRESSÃO TECIDUAL(Ubíquo)
Linfócitos
58.3 TPM
Tireoide
57.7 TPM
Útero
55.8 TPM
Cervix Endocervix
51.6 TPM
Testículo
51.5 TPM
OUTRAS DOENÇAS (2)
Coffin-Siris syndrome 11Coffin-Siris syndrome
HGNC:11106UniProt:Q96GM5

Variantes genéticas (ClinVar)

1,416 variantes patogênicas registradas no ClinVar.

🧬 ARID1A: NM_006015.6(ARID1A):c.802C>T (p.Gln268Ter) ()
🧬 ARID1A: NM_006015.6(ARID1A):c.2162-1G>A ()
🧬 ARID1A: NM_006015.6(ARID1A):c.2402dup (p.Gln802fs) ()
🧬 ARID1A: NM_006015.6(ARID1A):c.1970_1971del (p.Leu657fs) ()
🧬 ARID1A: NM_006015.6(ARID1A):c.2251+1G>A ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 1,060 variantes classificadas pelo ClinVar.

689
212
159
Patogênica (65.0%)
VUS (20.0%)
Benigna (15.0%)
VARIANTES MAIS SIGNIFICATIVAS
SOX4: NM_003107.3(SOX4):c.332dup (p.Ala112fs) [Likely pathogenic]
BICRA: Single allele [Likely pathogenic]
ARID2: NM_152641.4(ARID2):c.1483_1489del (p.Val495fs) [Pathogenic]
SOX4: NM_003107.3(SOX4):c.468_469del (p.Asp156fs) [Pathogenic]
ARID1B: NM_001374828.1(ARID1B):c.3250del (p.Ala1084fs) [Pathogenic]

Diagnóstico

Os sinais que médicos procuram e os exames que confirmam

Carregando...

Tratamento e manejo

Remédios, cuidados de apoio e o que precisa acompanhar

Carregando informações de tratamento...

Onde tratar no SUS

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

🇧🇷 Atendimento SUS — Síndrome Coffin-Siris

🗺️

Selecione um estado ou use sua localização para ver resultados.

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

Nenhum ensaio clínico registrado para esta condição.

🧪 Está conduzindo uma pesquisa?
Divulgue para pacientes e familiares que acompanham esta doença.
Divulgar pesquisa →

Publicações mais relevantes

Timeline de publicações
255 papers (10 anos)
#1

Expanding the Coffin-Siris syndrome spectrum: genetic, dysmorphic, and endocrine findings in eight cases.

European journal of pediatrics2026 Mar 08

This study aims to expand the spectrum of Coffin-Siris syndrome (CSS), a rare and heterogeneous disorder, by thoroughly discussing its genetic, dysmorphic, and endocrine features through new cases and contributing to the literature. Eight patients who were referred to the genetics clinic with various complaints and subsequently diagnosed with CSS through microarray or clinical exome sequencing analyses were included in the study. The dysmorphic, genetic, and endocrine characteristics of eight genetically confirmed patients were evaluated. The patients, aged between 5 months and 6 years at the time of referral, comprised four females and four males. The most common reasons for referral were developmental delay and dysmorphic features. All patients exhibited varying degrees of dysmorphic facial features. Hypertrichosis, a typical feature of the syndrome, was present in five patients. Another characteristic finding was mild hypoplasia of the terminal fifth phalanges, observed in patients 1, 2, and 6. Consistent with this, mild/subtle hypoplasia and/or slight positional changes of the fifth fingernails were noted in these patients, rather than overt nail anomalies. In our study, eight variants were identified, two of which were novel. In our cohort, pathological short stature was observed in three patients, while hypothyroidism, transient hypercalcemia, cryptorchidism, and recurrent fractures were each identified in one patient. All three patients with short stature had delayed bone age with head circumference and BMI <  - 2 SDS. Seven patients were diagnosed with ARID1B-related CSS type 1, while one patient was diagnosed with SMARCA4-related CSS type 4. Among the eight findings across patients, two were deletion-type copy-number variations (CNVs) identified by microarray analysis, and six were sequence variants: two frameshift, two splice-site, one nonsense, and one synonymous. Seven variants were classified as pathogenic and one as likely pathogenic. Family studies confirmed that the variants were de novo and validated their clinical relevance.  CSS is a clinically and genetically heterogeneous syndrome. Patients may present with highly variable features, and typical signs of the syndrome may not be observed in all cases. This study expands the clinical spectrum of this rare syndrome and contributes to its genetic spectrum with the identification of new variants. • Coffin-Siris syndrome (CSS) is a clinically and genetically heterogeneous neurodevelopmental disorder most commonly caused by variants in SWI/SNF (BAF) complex genes (e.g., ARID1B, SMARCA4) and characterized by dysmorphic features, developmental delay, hypertrichosis, and fifth-digit/nail anomalies. • Endocrine and growth-related manifestations can occur in CSS, but their frequency and phenotypic range vary across cohorts and require individualized clinical follow-up. • This case series of eight genetically confirmed CSS patients (7 ARID1B, 1 SMARCA4) expands the phenotypic spectrum by detailing dysmorphic findings together with endocrine features including pathological short stature with delayed bone age, hypothyroidism, transient hypercalcemia, cryptorchidism, and recurrent fractures. • We identified eight pathogenic/likely pathogenic variants, including two novel variants, and highlight that fifth digit/nail involvement may be subtle (mild terminal fifth phalanx hypoplasia and minor fifth nail changes) rather than overt.

#2

Expanding the Phenotypic Spectrum Associated With Loss-of-Function SMARCA4 Variants to Eye Developmental Anomalies.

Clinical genetics2026 Jan 22

The SMARCA4 gene encodes a catalytic subunit of the BRG1/BRM-associated factor complex, which regulates gene expression through chromatin remodeling. Heterozygous missense variants in this gene have been linked to Coffin-Siris syndrome, characterized by intellectual development disorder and various congenital anomalies (distinctive facial features, hypoplastic fifth digits, and malformations of the heart and central nervous system), but it is not typically associated with structural eye anomalies. Truncating variants in SMARCA4 have been associated with rhabdoid tumors predisposition syndrome, a group of rare and aggressive tumors occurring predominantly in infancy. Through pangenomic analyses (whole-exome or whole-genome sequencing), we identified loss-of-function variants in SMARCA4 in three unrelated individuals with microphthalmia and/or coloboma. None of these individuals had a history of rhabdoid tumors; however, a regular oncological follow-up was established following the SMARCA4 variant identification. Systemic features observed in these individuals consisted of developmental delay and brain anomalies. However, their clinical presentation does not align with classic features of Coffin-Siris syndrome. Although eye development anomalies have occasionally been reported in individuals with a pathogenic variant in SMARCA4, no clear association has been established to date. The description of these three new individuals provides further evidence supporting the role of SMARCA4 in eye development and its likely involvement in structural eye malformations.

#3

Identification of novel variants in the ARID1B gene causing Coffin-Siris syndrome.

European journal of pediatrics2026 Jan 17

Coffin-Siris Syndrome (CSS) is a neurodevelopmental disorder caused by variants in genes encoding BRG1- and BRM-associated factor (BAF) chromatin-remodeling complex. ARID1B gene variants are the most common cause of CSS. This study aimed to identify novel pathogenic ARID1B variants in patients clinically diagnosed with CSS and to explore their pathogenic role. In this study, eight patients clinically diagnosed with CSS were enrolled, and whole exome sequencing (WES) was performed to identify potential pathogenic variants. Heterozygous variants in the ARID1B gene were identified in six patients, including one previously reported pathogenic nonsense variant and five novel pathogenic truncating variants. The combined annotation-dependent depletion (CADD) scores of the five novel variants were significantly above the mutation significance cutoff (MSC), suggesting their potential pathogenicity. According to the guidelines of the American College of Medical Genetics and Genomics (ACMG), these five novel variants were classified as pathogenic. Our findings add five novel variants to the list of known pathogenic variants of the ARID1B gene. This study further clarifies an enhanced connection between ARID1B gene variants and CSS and expands the variant spectrum of CSS. • Coffin-Siris syndrome (CSS) is a rare neurodevelopmental disorder characterized by developmental delay, intellectual disability, and hypoplasia of the fifth digits or nails. • Pathogenic variants in genes encoding subunits of the BAF chromatin-remodeling complex are the major genetic causes of CSS, with ARID1B being the most frequently mutated gene, and most variants of which are truncating and lead to haploinsuffucuency. • Five novel heterozygous truncating variants in ARID1B were identified in eight patients clinically diagnosed with Coffin-Siris syndrome. • All novel variants showed high CADD scores and were classified as pathogenic according to ACMG guidelines.

#4

Discovery of novel candidate genes for congenital diaphragmatic hernia via whole exome sequencing.

Pediatrics international : official journal of the Japan Pediatric Society2026

Congenital diaphragmatic hernia (CDH) is a developmental anomaly associated with high mortality and morbidity, primarily attributed to accompanying pulmonary hypoplasia. Genetic factors are crucial in the etiology and pathogenesis of CDH, with various copy number variations (CNVs) and gene sequence variants implicated in this malformation. Previous studies have underscored the importance of retinoic acid (RA) signaling pathways and related genes. Nonetheless, the complexity of diaphragmatic development involving cell migration, cytoskeleton organization, and myogenesis suggests that candidate CDH genes extend beyond the RA pathway. To explore novel candidate gene variants and their roles in CDH, we performed whole exome sequencing (WES) in CDH-affected fetuses. Following the evaluation of chromosome and array-CGH analyses, 17 CDH cases with normal results in our cohort were subjected to WES. Trio-WES was conducted on eight fetuses, while solo-WES was applied to the remaining nine cases. The identified variants were validated and subjected to segregation analysis via Sanger sequencing. Bioinformatic analysis revealed novel potentially pathogenic variants not only in six genes previously known to be associated with CDH (NR2F2, ZFPM2, ARID1A, CREBBP, PLAT, and RARB) but also in nine additional genes (COL11A1, NEIL2, PCSK5, RBM8A, STAB2, SETD5, TAF4, ZBTB38, and ZNF423) that, based on their functions, database entries, and literature, may be considered candidate genes for CDH. Our findings reinforce that no single gene or variant is responsible for the majority of CDH cases, and also demonstrated the effectiveness of WES in identifying novel candidate genes and variants that contribute to CDH etiology.

#5

Unusual Recombinant Chromosome 6 Derived From a Parental Rearrangement With Complex Paracentric Inversions.

American journal of medical genetics. Part A2026 Mar 08

Complex chromosomal rearrangements (CCRs) are structural variants involving multiple breakpoints. Among these, intrachromosomal balanced CCRs containing inversions pose significant diagnostic and interpretative challenges, as conventional cytogenetic methods including G-banded karyotype, FISH, and chromosomal microarray lack the resolution needed to determine their structural complexity. Paracentric inversions are traditionally associated with a negligible risk of a viable unbalanced offspring. Here, we describe a familial intrachromosomal rearrangement comprising a complex paracentric inversion of chromosome 6q, transmitted across multiple generations, that resulted in five affected children with recombinant chromosomes harboring reciprocal interstitial gains and losses on 6q. High-resolution optical genome mapping revealed a ~75 Mb parental balanced CCR, formed by multiple sequential paracentric inversions that contain a single ~13 Mb correctly oriented segment. Bias meiotic recombination between homologous chromosomes 6 within this segment is responsible for recurrent unbalanced products resembling recombinant chromosomes typically associated with pericentric inversions. These findings challenge the prevailing assumption that paracentric inversions rarely result in viable recombinant chromosomes and demonstrate how complex chromosomal architecture can directly influence meiotic behavior and reproductive outcomes. Our study underscores the importance of high-resolution genomic technologies for accurate diagnosis, interpretation of a molecular mechanism, and reproductive risk assessment in carriers of CCR.

Publicações recentes

Ver todas no PubMed

📚 EuropePMC225 artigos no totalmostrando 195

2026

Expanding the Coffin-Siris syndrome spectrum: genetic, dysmorphic, and endocrine findings in eight cases.

European journal of pediatrics
2026

Unusual Recombinant Chromosome 6 Derived From a Parental Rearrangement With Complex Paracentric Inversions.

American journal of medical genetics. Part A
2026

A novel variant in ARID2 causes Coffin-Siris syndrome 6 with liver cirrhosis.

Gene
2026

Chromatin remodelling subunit SMARCB1 is implicated in dendrite development and complex brain functions.

Acta neuropathologica communications
2026

Double Lacrimal Puncta in a Case of Coffin-Siris Syndrome.

Ophthalmic plastic and reconstructive surgery
2026

Beyond Neurodevelopmental Delay: BICRA-Related Coffin-Siris Syndrome 12 with Severe Intestinal Dysmotility and Recurrent Pneumothorax.

Genes
2026

Expanding the Phenotypic Spectrum Associated With Loss-of-Function SMARCA4 Variants to Eye Developmental Anomalies.

Clinical genetics
2026

Identification of novel variants in the ARID1B gene causing Coffin-Siris syndrome.

European journal of pediatrics
2026

Clinical and Genetic Analysis of SMARCC2-Related Diseases in Three Chinese Patients.

Molecular genetics &amp; genomic medicine
2026

Discovery of novel candidate genes for congenital diaphragmatic hernia via whole exome sequencing.

Pediatrics international : official journal of the Japan Pediatric Society
2025

Prenatal Diagnosis of 6q Terminal Deletion Associated with Coffin-Siris Syndrome: Phenotypic Delineation and Review.

Genes
2025

Long-read sequencing identifies a novel de novo inversion in SMARCC2 in a pediatric patient with Coffin-siris syndrome 8: a case report.

BMC medical genomics
2025

Optic nerve hypoplasia/dysplasia in Coffin-Siris syndrome: a case series.

Journal of AAPOS : the official publication of the American Association for Pediatric Ophthalmology and Strabismus
2025

A Novel Variant in the BICRA Gene, Expanding the Phenotype: A Case Report.

Case reports in genetics
2025

ARID1A gene variants and fetal hydrocephalus: First evidence of mRNA decay escape.

European journal of medical genetics
2025

Case report: Surgical disconnection for medically refractory epilepsy in ARID1B-related Coffin-Siris syndrome.

Epilepsy &amp; behavior reports
2025

Case Report: Observation of early-onset high myopia with fundus tessellation changes in Coffin-Siris syndrome 9 (CSS9) and literature review.

Frontiers in pediatrics
2025

A Novel Variant of ARID1B-Related Coffin-Siris Syndrome in a Saudi Girl: A Case Report.

Cureus
2025

A Coffin-Siris syndrome-associated mutation modeled in Caenorhabditis elegans affects multiple developmental processes.

G3 (Bethesda, Md.)
2025

SMARCB1-related schwannomatosis and other SMARCB1-associated phenotypes: clinical spectrum and molecular pathogenesis.

Familial cancer
2025

Delayed diagnosis of glaucoma in Coffin-Siris syndrome.

American journal of ophthalmology case reports
2025

Identification of variants in SWI/SNF complex genes associated with neurodevelopmental disorders.

Frontiers in genetics
2025

[Two cases of Coffin-Siris syndrome type 3 caused by de novoSMARCB1 gene mutations].

Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics
2025

Evaluating the consistency of SMARCB1 variant classification and assertions of genotype-phenotype relationships in ClinVar.

Cancer genetics
2025

Inflammatory arthritis as an atypical manifestation of Coffin-Siris syndrome linked to a novel ARID1B variant.

Clinical and experimental rheumatology
2024

SMARCA4-related Coffin-Siris syndrome in newborn: a case report and literature review.

Frontiers in pediatrics
2025

Next Generation Phenotyping and Synthetic Faces in Coffin Siris Syndrome.

Clinical genetics
2024

ARID1B-related disorder in 87 adults: Natural history and self-sustainability.

Genetics in medicine open
2024

A novel mutation in SMARCB1 associated with adult Coffin-Siris syndrome and meningioma.

Acta biochimica et biophysica Sinica
2025

Coffin-Siris Syndrome and Unusual Angiogenic Profiles in Pregnancy: A Case Study Emphasizing Caution in Interpreting a Very Low sFlt-1/PlGF Ratio.

American journal of medical genetics. Part A
2026

BILATERAL MACULAR DYSPLASIA IN COFFIN-SIRIS SYNDROME.

Retinal cases &amp; brief reports
2024

A rare Coffin-Siris syndrome induced by SOX11: a de novo nonsense variant of short stature.

BMC medical genomics
2024

Novel Missense Variant in the SMARCD1 Gene as the Cause of Coffin-Siris Syndrome 11 in a Fetus With Ambiguous Genitalia and Multiple Dysmorphic Features.

Prenatal diagnosis
2025

Microduplications of ARID1A and ARID1B cause a novel clinical and epigenetic distinct BAFopathy.

Genetics in medicine : official journal of the American College of Medical Genetics
2024

[Genetic analysis of a fetus with Coffin-Siris syndrome 2 due to a novel variant of ARID1A gene].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
2024

The overlapping of phenotypes in Wiedemann-Steiner, Kleefstra and Coffin-Siris syndromes: a study of eleven patients.

Italian journal of pediatrics
2025

Expanding the Spectrum of Endocrine Abnormalities Associated With SOX11-related Disorders.

The Journal of clinical endocrinology and metabolism
2024

More than three years' treatment response of recombinant human growth hormone in a patient with Coffin-Siris syndrome 7.

Endokrynologia Polska
2024

ARID1A-BAF coordinates ZIC2 genomic occupancy for epithelial-to-mesenchymal transition in cranial neural crest specification.

American journal of human genetics
2024

PHF6 cooperates with SWI/SNF complexes to facilitate transcriptional progression.

Nature communications
2024

Coffin-Siris Syndrome and SMARCB1 Mutation Presenting With Schwannomatosis: A Case Report and Literature Review.

Cureus
2024

The missing link: ARID1B non-truncating variants causing Coffin-Siris syndrome due to protein aggregation.

Human genetics
2024

SEM-2/SoxC regulates multiple aspects of C. elegans postembryonic mesoderm development.

bioRxiv : the preprint server for biology
2025

First report of Coffin-Siris Syndrome with SMARCB1 variant, normal intelligence and mild selective neuropsychological deficits: A case report and literature review.

The Clinical neuropsychologist
2024

Arid1b haploinsufficiency in pyramidal neurons causes cellular and circuit changes in neocortex but is not sufficient to produce behavioral or seizure phenotypes.

bioRxiv : the preprint server for biology
2024

Nail dysplasia and digital hypoplasia ‒ Coffin-Siris syndrome.

Anais brasileiros de dermatologia
2024

A novel BICRA variant causing Coffin-Siris Syndrome.

Minerva medica
2024

Short Report: 10-year follow-up of a boy with ARID1B-related disorder. Early intervention, longitudinal dimensional phenotype, brain imaging and outcome.

Research in developmental disabilities
2025

Coffin-Siris syndrome: A case report and dental findings.

International journal of paediatric dentistry
2024

ARID1B maintains mesenchymal stem cell quiescence via inhibition of BCL11B-mediated non-canonical Activin signaling.

Nature communications
2024

De novo variation in ARID1B gene causes Coffin-Siris syndrome 1 in a Chinese family with excessive early-onset high myopia.

BMC medical genomics
2024

DNA methylation analysis in patients with neurodevelopmental disorders improves variant interpretation and reveals complexity.

HGG advances
2024

Expanding the clinical spectrum of Coffin-Siris syndrome with anorectal malformations: Case report and review of the literature.

European journal of medical genetics
2024

Coffin-Siris syndrome and apneas. Comment on "Coffin-Siris syndrome and delayed emergence-Is this an unusual or unknown anesthetic complication? Prabhakar P, Chandran SD, Tembhurne SA, Mathew A, Rai E. Pediatr Anesth. 2024; 00: 1-2. Doi: 10.1111/pan.14892".

Paediatric anaesthesia
2024

DPF2-related Coffin-Siris syndrome type 7 in two generations.

European journal of medical genetics
2024

Identification of a novel de novo mutation in SOX4 for syndromic tooth agenesis.

Clinical oral investigations
2024

Identification of a novel phenotype of external ear deformity related to Coffin-Siris syndrome-9 and literature review.

American journal of medical genetics. Part A
2024

Coffin-Siris syndrome and delayed emergence-Is this an unusual or unknown anesthetic complication?

Paediatric anaesthesia
2023

Oral and dental abnormalities in Coffin Siris syndrome : A new case report.

La Tunisie medicale
2024

Protein destabilization underlies pathogenic missense mutations in ARID1B.

Nature structural &amp; molecular biology
2024

Treatment of psychiatric comorbidities and interaction patterns in Coffin-Siris syndrome: A case report of a 4-year-old girl.

Clinical case reports
2024

ARID2, a milder cause of Coffin-Siris Syndrome? Broadening the phenotype with 17 additional individuals.

American journal of medical genetics. Part A
2023

Coffin-Siris Syndrome: Case Series of Three Patients and a Novel ARID2 Variant.

Annals of clinical and laboratory science
2024

[Genetic analysis of two children with Coffin-Siris syndrome due to variants of ARID1B gene].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
2024

Delineation of the adult phenotype of Coffin-Siris syndrome in 35 individuals.

Human genetics
2024

Prenatal Coffin-Siris Syndrome: Expanding the Phenotypic and Genotypic Spectrum of the Disease.

Pediatric and developmental pathology : the official journal of the Society for Pediatric Pathology and the Paediatric Pathology Society
2023

Autism spectrum disorder and Coffin-Siris syndrome-Case report.

Frontiers in psychiatry
2023

Microspherophakic Angle Closure Glaucoma in a Patient with Coffin-Siris Syndrome: Case Report.

The application of clinical genetics
2023

Integration of EpiSign, facial phenotyping, and likelihood ratio interpretation of clinical abnormalities in the re-classification of an ARID1B missense variant.

American journal of medical genetics. Part C, Seminars in medical genetics
2023

Recurrence of ARID1B -related Coffin-Siris Syndrome by possible gonadal mosaicism.

Clinical dysmorphology
2024

Pathogenic variants in SOX11 mimicking Pitt-Hopkins syndrome phenotype.

Clinical genetics
2023

Elucidating the clinical and molecular spectrum of SMARCC2-associated NDD in a cohort of 65 affected individuals.

Genetics in medicine : official journal of the American College of Medical Genetics
2023

Abnormal chromatin remodeling caused by ARID1A deletion leads to malformation of the dentate gyrus.

Cell death and differentiation
2023

First-trimester prenatal diagnosis of Coffin-Siris syndrome-related congenital diaphragmatic hernia: The role of exome sequencing in determining genetic etiology.

Congenital anomalies
2023

A de novo variant of BICRA results in Coffin-Siris syndrome 12.

Molecular genetics &amp; genomic medicine
2023

Onychodystrophy with Multiple Epiphyseal Dysplasia: Literature Review.

Skin appendage disorders
2023

Integrating whole-genome sequencing and transcriptomic findings in the diagnosis and management of Coffin-Siris syndrome.

Brain &amp; development
2023

Anesthetic management in a child with Coffin Siris syndrome.

Journal of anaesthesiology, clinical pharmacology
2023

Coffin-Siris syndrome and cancer susceptibility.

Genetics in medicine open
2023

Pigmentation abnormalities in Coffin-Siris syndrome.

Clinical genetics
2023

Facial analytics based on a coordinate extrapolation system (zFACE) for morphometric phenotyping of developing zebrafish.

Disease models &amp; mechanisms
2023

A newborn with coffin-siris syndrome.

JPMA. The Journal of the Pakistan Medical Association
2023

Genetic and genomic analyses of Drosophila melanogaster models of chromatin modification disorders.

Genetics
2023

Novel Variants of SOX4 in Patients with Intellectual Disability.

International journal of molecular sciences
2023

ARID2, a rare cause of Coffin-Siris syndrome: A novel microdeletion at 12q12q13.11 causing severe short stature and literature review.

American journal of medical genetics. Part A
2023

Discovering a new part of the phenotypic spectrum of Coffin-Siris syndrome in a fetal cohort.

Genetics in medicine : official journal of the American College of Medical Genetics
2023

Recommending revised hepatoblastoma surveillance in children with a pathogenic ARID1A variant. Reply to "Cancer in ARID1A-Coffin-Siris syndrome: Review and report of a child with hepatoblastoma" by Cárcamo et al. 2022.

European journal of medical genetics
2022

Growth Hormone Deficiency due to p.(Gln467Argfs*64) Mutation in the ARID1B Gene in a Girl with Coffin-Siris Syndrome.

Molecular syndromology
2023

Occurrence of sotos syndrome and coffin-siris syndrome in a family.

Pediatrics and neonatology
2022

Coffin-Siris syndrome: Clinical description of two cases.

Clinical case reports
2023

High molecular diagnostic yields and novel phenotypic expansions involving syndromic anorectal malformations.

European journal of human genetics : EJHG
2023

Establishment of an induced pluripotent stem cell (iPSC) line SDQLCHi045-A from peripheral blood mononuclear cells of a patient with Coffin-Siris syndrome 1 carrying a mutation in ARID1B gene.

Stem cell research
2022

[Diagnosis a fetus with Coffin-Siris syndrome due to variant of SMARCA4 gene by whole exome sequencing].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
2023

Identification of a novel BICRA variant leading to the newly described Coffin-Siris syndrome 12.

Brain &amp; development
2023

Congenital diaphragmatic hernia in Coffin Siris syndrome: Further evidence from two cases.

American journal of medical genetics. Part A
2022

Acetate supplementation restores cognitive deficits caused by ARID1A haploinsufficiency in excitatory neurons.

EMBO molecular medicine
2023

Two SOX11 variants cause Coffin-Siris syndrome with a new feature of sensorineural hearing loss.

American journal of medical genetics. Part A
2022

Three Novel ARID1B Variations in Coffin-Siris Syndrome Patients.

Neurology India
2023

Next-generation sequencing reanalysis identifies Coffin-Siris syndrome with an initial diagnosis of hypertrophic cardiomyopathy.

Pediatrics and neonatology
2023

Epilepsy in Coffin-Siris syndrome: A report from the international CSS registry and review of the literature.

American journal of medical genetics. Part A
2022

Pituitary hypoplasia and growth hormone deficiency in a patient with Coffin-Siris syndrome and severe short stature: case report and literature review.

Archive of clinical cases
2022

Severe coarctation of the aorta, developmental delay, and multiple dysmorphic features in a child with SMAD6 and SMARCA4 variants.

European journal of medical genetics
2022

"Cancer in ARID1A-Coffin-Siris syndrome: Review and report of a child with hepatoblastoma".

European journal of medical genetics
2022

Short stature and melanocytic nevi in a girl with ARID1B-related Coffin-Siris syndrome: a case report.

BMC pediatrics
2022

Identification and functional analysis of novel SOX11 variants in Chinese patients with Coffin-Siris syndrome 9.

Frontiers in genetics
2022

[Clinical features and genetic analysis of two Chinese patients with Coffin Siris syndrome-1].

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

A novel nonsense variant in ARID1B causing simultaneous RNA decay and exon skipping is associated with Coffin-Siris syndrome.

Human genome variation
2022

ARID2, a Rare Cause of Coffin-Siris Syndrome: A Clinical Description of Two Cases.

Frontiers in pediatrics
2022

Prenatal diagnosis of Coffin-Siris syndrome: What are the fetal features?

Prenatal diagnosis
2022

Evidence for an association between Coffin-Siris syndrome and congenital diaphragmatic hernia.

American journal of medical genetics. Part A
2022

On the Interaction Between SMARCAL1 and BRG1.

Frontiers in cell and developmental biology
2022

Neurocognitive, behavioral and socio-adaptive functioning assessment in a case of Coffin-Siris syndrome: A holistic approach/perspective beyond the identification of the disorder.

Journal of pediatric rehabilitation medicine
2022

Cochlear nerve deficiency in SOX11-related Coffin-Siris syndrome.

American journal of medical genetics. Part A
2022

A novel intragenic DPF2 deletion identified by genome sequencing in an adult with clinical features of Coffin-Siris syndrome.

American journal of medical genetics. Part A
2022

De Novo SMARCC2 Variant in a Chinese Woman with Coffin-Siris Syndrome 8: a Case Report with Mild Intellectual Disability and Endocrinopathy.

Journal of molecular neuroscience : MN
2022

Coffin-Siris syndrome in two chinese patients with novel pathogenic variants of ARID1A and SMARCA4.

Genes &amp; genomics
2022

[Analysis of ARID1B gene variants in two Chinese pedigrees with Coffin-Siris syndrome].

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

Neurodevelopmental disorders: 2022 update.

Free neuropathology
2022

Late-onset hypertension in a child with growth retardation: Answers.

Pediatric nephrology (Berlin, Germany)
2022

Expanding the phenotype associated with SMARCC2 variants: a fetus with tetralogy of Fallot.

BMC medical genomics
2022

Consolidation of the clinical and genetic definition of a SOX4-related neurodevelopmental syndrome.

Journal of medical genetics
2021

Language Impairments in Individuals With Coffin-Siris Syndrome.

Frontiers in neuroscience
2024

Frameshift Variant in ARID2 in a Chilean Individual with Coffin-Siris Syndrome Phenotype.

Journal of pediatric genetics
2022

Prenatal presentation of multiple anomalies associated with haploinsufficiency for ARID1A.

European journal of medical genetics
2022

Eyelash trichomegaly: a systematic review of acquired and congenital aetiologies of lengthened lashes.

Journal of the European Academy of Dermatology and Venereology : JEADV
2022

Retrospective analysis of a clinical exome sequencing cohort reveals the mutational spectrum and identifies candidate disease-associated loci for BAFopathies.

Genetics in medicine : official journal of the American College of Medical Genetics
2022

Further supporting SMARCC2-related neurodevelopmental disorder through exome analysis and reanalysis in two patients.

American journal of medical genetics. Part A
2022

Non-occlusive mesenteric ischemia in a toddler with 6q25 microdeletion syndrome.

Pediatrics international : official journal of the Japan Pediatric Society
2022

Novel Variants of the SMARCA4 Gene Associated with Autistic Features Rather Than Typical Coffin-Siris Syndrome in Eight Chinese Pediatric Patients.

Journal of autism and developmental disorders
2021

Identification of de novo mutations for ARID1B haploinsufficiency associated with Coffin-Siris syndrome 1 in three Chinese families via array-CGH and whole exome sequencing.

BMC medical genomics
2021

Inability to switch from ARID1A-BAF to ARID1B-BAF impairs exit from pluripotency and commitment towards neural crest formation in ARID1B-related neurodevelopmental disorders.

Nature communications
2021

Epilepsy features in ARID1B-related Coffin-Siris syndrome.

Epileptic disorders : international epilepsy journal with videotape
2021

Phenotypic and molecular spectra of patients with switch/sucrose nonfermenting complex-related intellectual disability disorders in Korea.

BMC medical genomics
2021

SMARCA4: Implications of an Altered Chromatin-Remodeling Gene for Cancer Development and Therapy.

Molecular cancer therapeutics
2021

Magnetically Controlled Growing Rods for Early Scoliosis Treatment in Coffin-Siris Syndrome: Case Report and Literature Review.

The Iowa orthopaedic journal
2021

Chromoanagenesis Event Underlies a de novo Pericentric and Multiple Paracentric Inversions in a Single Chromosome Causing Coffin-Siris Syndrome.

Frontiers in genetics
2021

A Case Series of Familial ARID1B Variants Illustrating Variable Expression and Suggestions to Update the ACMG Criteria.

Genes
2021

CHARGE syndrome and related disorders: a mechanistic link.

Human molecular genetics
2021

Genotype-Phenotype Correlations in 208 Individuals with Coffin-Siris Syndrome.

Genes
2021

Rehabilitation in a rare case of coffin-siris syndrome with major cognitive and behavioural disorders.

Journal of pediatric rehabilitation medicine
2021

Metacarpophalangeal profile pattern analysis in a further patient with a novel ARID1B variant.

Congenital anomalies
2021

Two Siblings with Kaufman Oculocerebrofacial Syndrome Resembling Oculoauriculovertebral Spectrum.

Molecular syndromology
2021

Chromatin remodeler Arid1a regulates subplate neuron identity and wiring of cortical connectivity.

Proceedings of the National Academy of Sciences of the United States of America
2021

Novel ARID1B variant inherited from somatogonadal mosaic mother in siblings with Coffin-Siris syndrome 1.

Experimental and therapeutic medicine
2021

Generation of an iPSC line (CRICKi001-A) from an individual with a germline SMARCA4 missense mutation and autism spectrum disorder.

Stem cell research
2022

Maternal transmission of a mild Coffin-Siris syndrome phenotype caused by a SOX11 missense variant.

European journal of human genetics : EJHG
2022

Clinical and molecular features of idiopathic hypogonadotropic hypogonadism in Taiwan: A single center experience.

Journal of the Formosan Medical Association = Taiwan yi zhi
2021

Genotype and phenotype in 18 Chinese patients with Coffin-Siris syndrome.

American journal of medical genetics. Part A
2021

Neuroanatomy and behavior in mice with a haploinsufficiency of AT-rich interactive domain 1B (ARID1B) throughout development.

Molecular autism
2021

The Evolutionary Conserved SWI/SNF Subunits ARID1A and ARID1B Are Key Modulators of Pluripotency and Cell-Fate Determination.

Frontiers in cell and developmental biology
2021

A boy with Coffin-Siris syndrome with a novel frameshift mutation in ARID1B.

Neuro endocrinology letters
2021

Rhabdoid Tumor Predisposition Syndrome: From Clinical Suspicion to General Management.

Frontiers in oncology
2021

Current recommendations for clinical surveillance and genetic testing in rhabdoid tumor predisposition: a report from the SIOPE Host Genome Working Group.

Familial cancer
2021

Temporal bone dysplasia in Coffin-Siris syndrome.

BMJ case reports
2022

Clinical exome sequencing data reveal high diagnostic yields for congenital diaphragmatic hernia plus (CDH+) and new phenotypic expansions involving CDH.

Journal of medical genetics
2021

First observation of secondary childhood glaucoma in Coffin-Siris syndrome: a case report and literature review.

BMC ophthalmology
2021

A Novel De Novo Heterozygous ARID1A Missense Variant Cluster in cis c.[5954C>G;6314C>T;6334C>T;6843G>C] causes a Coffin-Siris Syndrome.

Annals of laboratory medicine
2020

Multiple Developmental Defects in sox11a Mutant Zebrafish with Features of Coffin-Siris Syndrome.

International journal of biological sciences
2021

Association between ARID2 and RAS-MAPK pathway in intellectual disability and short stature.

Journal of medical genetics
2021

Coffin-Siris syndrome and epilepsy.

Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology
2020

Coffin-Siris Syndrome 4-Related Spectrum in a Young Woman Caused by a Heterozygous SMARCA4 Deletion Detected by High-Resolution aCGH.

Molecular syndromology
2020

Systemic and ocular manifestations of a patient with mosaic ARID1A-associated Coffin-Siris syndrome and review of select mosaic conditions with ophthalmic manifestations.

American journal of medical genetics. Part C, Seminars in medical genetics
2020

Growth charts for individuals with Coffin-Siris syndrome.

American journal of medical genetics. Part A
2020

Pure 9p duplication syndrome with aplasia of the middle phalanges of the fifth fingers.

European journal of medical genetics
2020

The variability of SMARCA4-related Coffin-Siris syndrome: Do nonsense candidate variants add to milder phenotypes?

American journal of medical genetics. Part A
2020

CRISPR/Cas9 mediated generation of human ARID1B heterozygous knockout hESC lines to model Coffin-Siris syndrome.

Stem cell research
2020

Koebner phenomenon of vitiligo associated with Coffin-Siris syndrome.

European journal of dermatology : EJD
2020

Coffin-Siris syndrome with bilateral macular dysplasia caused by a novel exonic deletion in ARID1B.

Congenital anomalies
2020

Discovering candidate imprinted genes and imprinting control regions in the human genome.

BMC genomics
2020

Coffin-Siris Syndrome-1: Report of five cases from Asian populations with truncating mutations in the ARID1B gene.

Journal of the neurological sciences
2020

Conserved roles of chromatin remodellers in cohesin loading onto chromatin.

Current genetics
2020

First Korean Case of Coffin-Siris Syndrome with a Novel Frameshift ARID1B Mutation.

Annals of clinical and laboratory science
2019

Recurrent SMARCB1 Mutations Reveal a Nucleosome Acidic Patch Interaction Site That Potentiates mSWI/SNF Complex Chromatin Remodeling.

Cell
2019

BRM: the core ATPase subunit of SWI/SNF chromatin-remodelling complex-a tumour suppressor or tumour-promoting factor?

Epigenetics &amp; chromatin
2020

A new missense mutation in DPF2 gene related to Coffin Siris syndrome 7: Description of a mild phenotype expanding DPF2-related clinical spectrum and differential diagnosis among similar syndromes epigenetically determined.

Brain &amp; development
2019

Phenotypic Variation between Monochorionic Diamniotic Twins with Coffin-Siris Syndrome.

Journal of pediatric genetics
2020

SMARCE1-related Coffin-Siris Syndrome: Case report and otolaryngologic manifestations of the syndrome.

International journal of pediatric otorhinolaryngology
2019

Genome-Wide Analysis of the Nucleosome Landscape in Individuals with Coffin-Siris Syndrome.

Cytogenetic and genome research
2019

De novo splice site variant of ARID1B associated with pathogenesis of Coffin-Siris syndrome.

Molecular genetics &amp; genomic medicine
2019

Genetic abnormalities in a large cohort of Coffin-Siris syndrome patients.

Journal of human genetics
2020

Striking phenotypic overlap between Nicolaides-Baraitser and Coffin-Siris syndromes in monozygotic twins with ARID1B intragenic deletion.

European journal of medical genetics
2019

Mutations in SMARCB1 and in other Coffin-Siris syndrome genes lead to various brain midline defects.

Nature communications
2019

Inflammatory Arthritis as a Possible Feature of Coffin-Siris Syndrome.

Pediatrics
2019

Expanding the phenotypic spectrum associated with DPF2: A new case report.

American journal of medical genetics. Part A
2019

A case of Coffin-Siris syndrome with severe congenital heart disease and a novel SMARCA4 variant.

Cold Spring Harbor molecular case studies
2019

Significant contribution of intragenic deletions to ARID1B mutation spectrum.

Genetics in medicine : official journal of the American College of Medical Genetics
2019

A novel human stem cell model for Coffin-Siris syndrome-like syndrome reveals the importance of SOX11 dosage for neuronal differentiation and survival.

Human molecular genetics
2019

Retinal dystrophy in an individual carrying a de novo missense variant of SMARCA4.

Molecular genetics &amp; genomic medicine
2019

Corpus callosum metrics predict severity of visuospatial and neuromotor dysfunctions in ARID1B mutations with Coffin-Siris syndrome.

Psychiatric genetics
2019

Raised intra-ocular pressure in the setting of Coffin-Siris syndrome.

Eye (London, England)
2019

A Syndromic Neurodevelopmental Disorder Caused by Mutations in SMARCD1, a Core SWI/SNF Subunit Needed for Context-Dependent Neuronal Gene Regulation in Flies.

American journal of human genetics
2019

Patient with anomalous skin pigmentation expands the phenotype of ARID2 loss-of-function disorder, a SWI/SNF-related intellectual disability.

American journal of medical genetics. Part A
2019

Histone 4 Lysine 20 Methylation: A Case for Neurodevelopmental Disease.

Biology
2019

De Novo SOX4 Variants Cause a Neurodevelopmental Disease Associated with Mild Dysmorphism.

American journal of human genetics
2019

Vitiligo and melanocytic nevi: New findings in Coffin-Siris syndrome associated with ARID1 germline mutation.

JAAD case reports
Ver todos os 225 no EuropePMC

Associações

Organizações que acompanham esta doença — pra ter apoio e orientação

Ainda não temos associações cadastradas para Síndrome Coffin-Siris.

É de uma associação que acompanha esta doença? Fale com a gente →

Comunidades

Grupos ativos de quem convive com esta doença aqui no Raras

Ainda não existe comunidade no Raras para Síndrome Coffin-Siris

Pacientes, familiares e cuidadores se organizam em comunidades pra compartilhar experiências, fazer perguntas e se apoiar. Você pode ser o primeiro.

Tire suas dúvidas

Perguntas, dicas e experiências compartilhadas aqui na página

Participe da discussão

Faça login para postar dúvidas, compartilhar experiências e interagir com especialistas.

Fazer login

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. Expanding the Coffin-Siris syndrome spectrum: genetic, dysmorphic, and endocrine findings in eight cases.
    European journal of pediatrics· 2026· PMID 41795723mais citado
  2. Expanding the Phenotypic Spectrum Associated With Loss-of-Function SMARCA4 Variants to Eye Developmental Anomalies.
    Clinical genetics· 2026· PMID 41568967mais citado
  3. Identification of novel variants in the ARID1B gene causing Coffin-Siris syndrome.
    European journal of pediatrics· 2026· PMID 41545737mais citado
  4. Discovery of novel candidate genes for congenital diaphragmatic hernia via whole exome sequencing.
    Pediatrics international : official journal of the Japan Pediatric Society· 2026· PMID 41454640mais citado
  5. Unusual Recombinant Chromosome 6 Derived From a Parental Rearrangement With Complex Paracentric Inversions.
    American journal of medical genetics. Part A· 2026· PMID 41795620mais citado
  6. Congenital hypothyroidism with preserved fifth digits in SMARCA4-related Coffin-Siris syndrome: a case report.
    AME Case Rep· 2026· PMID 41971929recente
  7. ARID1B regulates sphingolipid metabolism and myelin development via STAG2: Mechanistic insights into ARID1B-related coffin-siris syndrome.
    Cell Signal· 2026· PMID 41967622recente
  8. Intranasal exosome therapy in Coffin-Siris syndrome: Clinical evaluation of three children.
    Surg Neurol Int· 2026· PMID 41952703recente
  9. Primary congenital glaucoma in a patient with Coffin-siris syndrome type 1 due to an ARID1B mutation: a novel association.
    Ophthalmic Genet· 2026· PMID 41911953recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:1465(Orphanet)
  2. MONDO:0015452(MONDO)
  3. GARD:6124(GARD (NIH))
  4. Variantes catalogadas(ClinVar)
  5. Busca completa no PubMed(PubMed)
  6. Q2348105(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 Coffin-Siris
Compêndio · Raras BR

Síndrome Coffin-Siris

ORPHA:1465 · MONDO:0015452
Prevalência
<1 / 1 000 000
Casos
190 casos conhecidos
Herança
Autosomal dominant
CID-10
Q87.1 · Síndromes com malformações congênitas associadas predominantemente com nanismo
CID-11
Início
Antenatal, Neonatal
Prevalência
0.0 (Worldwide)
MedGen
UMLS
C0265338
EuropePMC
Wikidata
Papers 10a
DiscussaoAtiva

Nenhuma novidade ainda. O agente esta monitorando.

0membros
0novidades