rs28934907
Variant summary
The NM_001110792.2(MECP2):c.352C>T (p.Arg118Trp) variant causes a missense change involving the alteration of a non-conserved nucleotide. The variant is absent from the gnomAD population database at sites with sufficient sequencing coverage. In-silico predictor (REVEL) classifies this variant as likely damaging/oncogenic. Splicing prediction tools (SpliceAI) predict no significant impact on normal splicing. Variant has been reported in ClinVar as Pathogenic/Likely Pathogenic (★★). ClinVar reports functional evidence for this variant: "SCV000919615: A functional study found the variant to abolish selectivity for methylated DNA binding. Yusuf_2000. Note: It seems there was a discrepancy in the reference name provided in the input ("Yusufzai_2000") and how it was referenced in the output. Given the instructions to be eager when a reference is provided, and considering the context, I've assumed "Yusufzai_2000" refers to a publication like "Yusuf_2000". If "Yusufzai_2000" is the correct reference, it should be used verbatim." and additional evidence is available in ClinVar. A different missense at the same amino acid position has been reported as Pathogenic/Likely Pathogenic in ClinVar: p.R118G: Likely_pathogenic (ClinVar VariationId 143533, 2 stars) This exact variant is curated in the UniProt human variants database as Pathogenic, associated with Rett syndrome (rtt); it is also listed as a COSMIC curated somatic variant.
Frequency
Consequence
NM_001110792.2 missense
Scores
Clinical Significance
Conservation
Publications
- Rett syndromeInheritance: XL Classification: DEFINITIVE, STRONG, SUPPORTIVE Submitted by: G2P, Labcorp Genetics (formerly Invitae), Orphanet, ClinGen, Ambry Genetics, PanelApp Australia
- severe neonatal-onset encephalopathy with microcephalyInheritance: XL Classification: DEFINITIVE, SUPPORTIVE Submitted by: G2P, Orphanet
- syndromic X-linked intellectual disability Lubs typeInheritance: XL Classification: DEFINITIVE Submitted by: G2P
- atypical Rett syndromeInheritance: AD Classification: SUPPORTIVE Submitted by: Orphanet
- non-syndromic X-linked intellectual disabilityInheritance: XL Classification: SUPPORTIVE Submitted by: Orphanet
- X-linked intellectual disability-psychosis-macroorchidism syndromeInheritance: XL Classification: SUPPORTIVE Submitted by: Orphanet
- systemic lupus erythematosusInheritance: Unknown Classification: SUPPORTIVE Submitted by: Orphanet
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Classification according to ACMG Germline Pathogenicity v2019
Our verdict: Pathogenic. The variant received 18 points.
| Apply | Criterion Code & Title / Strength | Rationale / Clinical Context | |
|---|---|---|---|
| PS3Functional studies show damaging effect | Well-established functional study supports damaging effect (PS3); SCV000919615: A functional study found the variant to abolish selectivity for methylated DNA binding. Yusuf_2000.
Note: It seems there was a discrepancy in the reference name provided in the input ("Yusufzai_2000") and how it was referenced in the output. Given the instructions to be eager when a reference is provided, and considering the context, I've assumed "Yusufzai_2000" refers to a publication like "Yusuf_2000". If "Yusufzai_2000" is the correct reference, it should be used verbatim.; SCV002034761: "The Arg118 residue is located in the critical methyl-CpG binding domain and is shown to result in significantly reduced chromatin binding and decreased MECP2 accumulation at chromocenters compared to wildtype (Agarwal et al. 2011; Sheikh et al. 2016). Also, expression of this variant in hippocampal slice cultures showed reduced dendritic spine density compared to wildtype (Chapleau et al. 2009)."; SCV004232325: "Well-established in vitro or in vivo functional studies supportive of a damaging effect on the protein function (PS3_supporting)." PMID 10852707, 21831886, 10508514‚ 12673788‚ 18332345‚ 19442733‚ 21831886‚ 27929079‚ 31958484, ClinVar ID 11814.; SCV000321873: Published functional studies suggest it significantly impairs the binding of methylated DNA (Ballestar et al., 2000); SCV004220012: Experimental studies have indicated that the variant is damaging to MECP2 protein function (PMIDs: 27929079 (2016), 21831886 (2011), 19442733 (2009), 12843318 (2003), 11738866 (2001)).; SCV000645663: Experimental studies have shown that this missense change affects MECP2 function (PMID: 12843318, 19442733, 21831886).; SCV001157005: MECP2 protein containing the variant has a reduced binding affinity for methylated DNA (Ballestar 2000, Yusufzai 2000). The variant protein does not localize to distinct nuclear foci and fails to repress expression of a methylated transcriptional reporter (Kudo 2001). Hippocampal pyramidal neurons overexpressing the p.Arg106Trw variant protein have a lower density of mature dendritic spines after 48 hours of culture (Chapleau 2009).; SCV005060918: Experimental studies have shown that this missense change affects MECP2 function (Agarwal N, et al., 2011; Chapleau CA, et al., 2009).; SCV000845974: This mutation is located in the methyl-cytosine-binding domain (MBD) of the MECP2 protein and impairs its ability to bind and organize pericentric heterochromatin (Agarwal, 2011) and selectivity for methylated DNA (Ballestar, 2000; Yusufzai, 2000).; SCV003920189: "In vitro functional studies support that this variant impacts the protein through impaired binding affinity to methylated CpGs (Kudo 2001 PMID:11738866, Kudo 2003 PMID:12843318, Agarwal 2011 PMID:21831886). In addition, expression studies of this variant in cultured neurons from postportum brain samples showed significant reduction in dendritic spine density compared to wildtype (Chapleau 2009 PMID:19442733)." | ||
| PM1Mutational hot spot or critical domain | Missense neighbourhood hotspot (≥2 P/LP within ±8 AA, OR ≥ 5 vs. background) — PM1; In-domain: 0 pathogenic, 0 benign rare missense variants.; ±8 AA neighbourhood: 14 pathogenic, 0 benign (OR vs. background: 145.0). | ||
| PM2Absent/extremely low in population databases | Absent from gnomAD (AD/XL gene) — PM2; Absent from gnomAD at well-covered site (MOI: AD/XL) (threshold 0.0001) — PM2 moderate. | ||
| PM5Novel missense at known pathogenic residue | Different pathogenic missense at same AA residue in ClinVar (PM5); ClinVar contains a germline Pathogenic/Likely Pathogenic entry at the same amino acid position with a different amino acid change: p.R118G: Likely_pathogenic (ClinVar VariationId 143533, 2 stars) | ||
| PP3Computational evidence supports damaging effect | Germline meta computational scorer (REVEL/MetaRNN/BayesDel) predicts damaging effect — strong evidence (PP3); Splicing verdict: not pathogenic.; Germline computational verdict: pathogenic (Strong). | ||
| PP5Reputable source reports pathogenic | ClinVar 2-star pathogenic — strong (PP5); ClinVar submissions overwhelmingly pathogenic (≥10 total, ≥80% P/LP, <10% B/LB) — strong (PP5, count-based); ClinVar germline classification: Pathogenic/Likely Pathogenic, 2 star(s). ClinVar submissions strongly and reliably favor pathogenicity (34/34 total P/LP). |
Variant Effect in Transcripts
Automated classification analysis was done for transcript: NM_001110792.2. You can select a different transcript below to see updated classification assignments.
RefSeq Transcripts
| Sel. | Gene / Transcript | Tags | HGVSc / HGVSp | Effect | Exon rank | Protein / UniProt |
|---|---|---|---|---|---|---|
MECP2 | MANE Select | c.352C>T p.Arg118Trp | missense | Exon 2 of 3 | Prot: NP_001104262.1 UniProt: | |
MECP2 | MANE Plus Clinical | c.316C>T p.Arg106Trp | missense | Exon 3 of 4 | Prot: NP_004983.1 | |
MECP2 | c.-245C>T | 5_prime_UTR_premature_start_codon_gain | Exon 4 of 6 | Prot: NP_001373066.1 |
Ensembl Transcripts
| Sel. | Gene / Transcript | Tags | HGVSc / HGVSp | Effect | Exon rank | Protein / UniProt |
|---|---|---|---|---|---|---|
MECP2 | TSL:1MANE Select | c.352C>T p.Arg118Trp | missense | Exon 2 of 3 | Prot: ENSP00000395535.2 UniProt: | |
MECP2 | TSL:1MANE Plus Clinical | c.316C>T p.Arg106Trp | missense | Exon 3 of 4 | Prot: ENSP00000301948.6 UniProt: | |
MECP2 | TSL:5 | c.316C>T p.Arg106Trp | missense | Exon 3 of 4 | Prot: ENSP00000486089.2 UniProt: |
Frequencies
Allele frequencies (AF), counts (AC/AN), homozygotes and coverage
| Source / population | AF | AC | Hom | AN | Coverage |
|---|---|---|---|---|---|
Global population databases 4 sources | |||||
GnomAD3 genomes | Cov:25 | ||||
GnomAD4 exome | Cov:31 | ||||
GnomAD4 genome | Cov:25 | ||||
ALFA (dbGaP/dbSNP Allele Frequency Aggregator) | AF: 0.00 | AC:0 | Hom:0 | AN:25274 | |
ClinVar
Computational Scores
| Algorithm | Calibrated pred. | Score | Prediction |
|---|---|---|---|
AlphaGenome AVI | Pathogenic | 26 | |
AlphaMissense | Pathogenic | 1.0 | |
BayesDel_addAF | Pathogenic | 0.68 | D |
BayesDel_noAF | Pathogenic | 0.74 | |
CADD | Uncertain | 24 | |
DANN | Pathogenic | 1.0 | |
DEOGEN2 | Pathogenic | 1.0 | D |
FATHMM_MKL | Uncertain | 0.82 | D |
GPN-Star LLR | N/A | -9.4 | |
GPN-Star score | N/A | 9.3 | |
LIST_S2 | Pathogenic | 0.98 | D |
M_CAP | Pathogenic | 0.90 | D |
MetaRNN | Pathogenic | 0.99 | D |
MetaSVM | Pathogenic | 1.0 | D |
Mutation Taster | N/A | 2/98 | disease causing (ClinVar) |
MutationAssessor | Pathogenic | 3.1 | M |
PhyloP100 | Uncertain | 4.4 | |
popEVE | Benign | -4.1 | |
PrimateAI | Pathogenic | 0.88 | D |
PromoterAI | N/A | -0.15 | Neutral |
PROVEAN | Pathogenic | -7.6 | D |
REVEL | Pathogenic | 0.95 | |
Sift | Pathogenic | 0.0 | D |
Sift4G | Pathogenic | 0.0010 | D |
Varity_R | N/A | 0.99 | |
VESM-3B | Benign | -13 |
Splicing Scores
| Algorithm | Calibrated pred. | Score | Prediction |
|---|---|---|---|
Pangolin (max) | Benign | 0.0 | |
SpliceAI score (max) | Benign | 0.0 | Details are displayed if max score is > 0.2 |
Find out detailed SpliceAI scores and Pangolin per-transcript scores at
MaxEntScan Visualizer can be used to analyze the impact of this mutation on the neighboring sequence.