MECP2 Gene Complete Identifier and Functional Mapping Reference

Provide a comprehensive cross-database identifier and functional mapping reference for human MECP2 — a definitive lookup resource covering: ### …

Provide a comprehensive cross-database identifier and functional mapping reference for human MECP2 — a definitive lookup resource covering: ### Section 1: Gene identifiers For human gene MECP2, list ALL gene-level database identifiers. Required: - HGNC ID and approved symbol - Ensembl gene ID (ENSG...) - NCBI Entrez Gene ID - OMIM gene/locus ID - Genomic location: chromosome, start position, end position, strand (GRCh38) ### Section 2: Transcript identifiers For human gene MECP2, list ALL transcript-level identifiers. Required: - Ensembl transcripts: ALL ENST IDs with biotype. Total count. - RefSeq transcripts: ALL NM_ mRNA accessions. Mark which is MANE Select. - CCDS IDs. - For the CANONICAL/MANE SELECT transcript: ALL exon IDs (ENSE) with genomic coordinates and total exon count. ### Section 3: Protein identifiers For human gene MECP2 protein product(s), list ALL protein-level identifiers. Required: - UniProt accessions: ALL entries (reviewed and unreviewed). Mark the canonical reviewed entry. - RefSeq protein: ALL NP_ accessions. - Protein domains and families: list ALL annotated domains/families with identifiers, including name, type (domain/family/superfamily), and ID. - Antibody availability: known antibody resources for the protein. ### Section 4: Structure For human gene MECP2 protein, list ALL structural data. Required: - Experimental structures: ALL PDB IDs. For each: experimental method (X-ray/NMR/Cryo-EM) and resolution. Total count. - Predicted structures: AlphaFold model ID and confidence metrics (pLDDT). ### Section 5: Cross-species orthologs For human gene MECP2, list orthologous genes in key model organisms. Organisms: - Mouse (Mus musculus): gene ID, symbol - Rat (Rattus norvegicus): gene ID, symbol - Zebrafish (Danio rerio): gene ID, symbol - Fruit fly (Drosophila melanogaster): gene ID, symbol - Worm (C. elegans): gene ID, symbol - Yeast (S. cerevisiae): gene ID, symbol ### Section 6: Clinical variants & AI predictions For human gene MECP2, summarize clinical variants and AI predictions. Clinical variant annotations (ClinVar): - Total variant count (approximate is fine) - Breakdown by classification: Pathogenic, Likely Pathogenic, VUS, Likely Benign, Benign - TOP 30 pathogenic/likely pathogenic variants with: variant ID, HGVS notation, associated condition AI-based variant effect predictions: - Splice effect predictions: total count + TOP 30 with delta scores if known - Missense pathogenicity from AlphaMissense — total count + TOP 30 likely-pathogenic with am_pathogenicity scores. ### Section 7: Pathways & Gene Ontology For human gene MECP2, list biological pathways and Gene Ontology annotations. Pathway membership: - ALL biological pathways this gene participates in, with pathway IDs and names - Total pathway count Gene Ontology: - Biological Process: count and TOP 20 terms with GO IDs - Molecular Function: count and TOP 20 terms with GO IDs - Cellular Component: count and TOP 20 terms with GO IDs ### Section 8: Protein interactions & networks For human gene MECP2 protein, summarize protein interactions and networks. Protein-protein interactions (STRING, IntAct, BioGRID, etc.): - Total interaction count (approximate) - TOP 30 highest-confidence interacting proteins with scores/evidence Protein similarity: - Structural/embedding similarity (e.g. Foldseek, ESM): TOP 20 similar proteins with scores - Sequence homology: TOP 20 homologous proteins with identity/similarity ### Section 9: Transcription factor regulatory data For human gene MECP2, summarize transcription factor regulatory data. If MECP2 is a transcription factor: - Downstream targets: total count + TOP 30 with regulation type (activates/represses) and evidence - DNA binding motifs from JASPAR — all known motif IDs and motif family classification. Regardless: - Upstream regulators: TFs that regulate MECP2 — names with evidence type (ChIP-seq / predicted / experimentally validated) If MECP2 is not a transcription factor, say so briefly and skip the downstream/motif sections. ### Section 10: Drug & pharmacology data For human gene MECP2 protein as a drug target, summarize pharmacology data. If MECP2 is a known drug target: - Targeting molecules: total count in ChEMBL/DrugBank + TOP 30 by development phase (molecule ID, name, mechanism, highest phase) - Clinical trials: TOP 20 involving drugs targeting this gene — trial ID, phase, status, intervention - Pharmacogenomics: known drug-gene interactions affecting drug response + dosing guidelines if any If MECP2 is not currently a drug target, say so briefly. ### Section 11: Expression profiles For human gene MECP2, summarize expression profiles. Tissue expression (GTEx, HPA, Bgee, etc.): - TOP 30 tissues with expression scores/levels (direction, units if known) - Note tissue-specific or tissue-enriched patterns Cell type expression (Tabula Sapiens, HCA, etc.): - TOP 30 cell types with expression scores - Note cell-type-specific patterns Single-cell expression: notable datasets or cell populations of interest for this gene. ### Section 12: Disease associations For human gene MECP2, summarize disease associations. Mendelian / monogenic disease: - Diseases caused by mutations in MECP2: disease name, disease ID (OMIM/Orphanet/Mondo), inheritance pattern, evidence level - Include all directly linked conditions Phenotype associations: - Clinical phenotypes associated with the gene (HPO terms where known) - TOP 30 phenotype terms with HPO IDs Complex-disease / GWAS: - Traits and diseases significantly associated via GWAS: trait name, variant, effect size, study where known - TOP 30 GWAS associations

MECP2

Executive summary

MECP2 (methyl-CpG-binding protein 2; HGNC:6990) is an X-linked epigenetic regulator that reads DNA methylation marks and recruits transcriptional corepressor complexes, making it essential for neuronal function and brain development. Loss-of-function mutations are the definitive cause of Rett syndrome (OMIM:312750), a severe progressive neurodevelopmental disorder, while MECP2 duplication causes a distinct X-linked dominant syndrome; together these represent the gene’s most critical clinical relevance. The protein is ubiquitously expressed across 299 conditions (average score 83.27/100), with highest levels in multiple brain regions and peripheral nervous tissue, consistent with its neurological disease burden. MECP2 sits at the center of a large interaction network (~3,950 STRING interactions) anchored by chromatin repressors HDAC1/2, SIN3A, and DNMT1, and it transcriptionally represses key neuronal targets including BDNF, GRIN2B, and GAD1. Clinically, ~900 ClinVar variants have been catalogued, with ~200 classified pathogenic or likely pathogenic, and AlphaMissense flags positions around residues 301–310 as among the highest-confidence pathogenic hotspots; however, no approved drugs target MECP2 directly, and all 6 ChEMBL compounds remain pre-clinical.

Gene identifiers

  • HGNC ID: HGNC:6990
  • Approved symbol: MECP2
  • Ensembl gene ID: ENSG00000169057
  • NCBI Entrez Gene ID: 4204
  • OMIM gene ID: 300005
  • Genomic location (GRCh38): Chromosome X, 154,021,573–154,137,103 (reverse strand)

Transcript identifiers

Ensembl Transcripts (Total: 30)

Transcript IDBiotype
ENST00000303391protein_coding
ENST00000369957nonsense_mediated_decay
ENST00000407218protein_coding
ENST00000415944protein_coding
ENST00000453960protein_coding
ENST00000460227protein_coding_CDS_not_defined
ENST00000463644protein_coding_CDS_not_defined
ENST00000481807retained_intron
ENST00000486506retained_intron
ENST00000488293protein_coding_CDS_not_defined
ENST00000496908protein_coding_CDS_not_defined
ENST00000611468protein_coding_CDS_not_defined
ENST00000625300protein_coding_CDS_not_defined
ENST00000626422protein_coding_CDS_not_defined
ENST00000627864protein_coding_CDS_not_defined
ENST00000628176protein_coding
ENST00000629277retained_intron
ENST00000630151protein_coding
ENST00000631210protein_coding_CDS_not_defined
ENST00000637467protein_coding_CDS_not_defined
ENST00000637533protein_coding_CDS_not_defined
ENST00000637791protein_coding_CDS_not_defined
ENST00000637917protein_coding
ENST00000638041protein_coding_CDS_not_defined
ENST00000674996nonsense_mediated_decay
ENST00000675526nonsense_mediated_decay
ENST00000675841retained_intron
ENST00000676382protein_coding_CDS_not_defined
ENST00000700484protein_coding_CDS_not_defined
ENST00000713611nonsense_mediated_decay

RefSeq mRNA Accessions (Human, 11 NM_ accessions)

AccessionStatusMANE Select
NM_001081979VALIDATEDNo
NM_001110792REVIEWEDYes
NM_001316337REVIEWEDNo
NM_001369391REVIEWEDNo
NM_001369392REVIEWEDNo
NM_001369393REVIEWEDNo
NM_001369394REVIEWEDNo
NM_001386137REVIEWEDNo
NM_001386138REVIEWEDNo
NM_001386139REVIEWEDNo
NM_004992REVIEWEDNo

CCDS IDs (2 total)

  • CCDS14741
  • CCDS48193 (MANE SELECT)

Exons for MANE SELECT Transcript ENST00000453960 (3 exons total)

Exon IDGenomic CoordinatesStrand
ENSE00001877797X:154097604-154097717
ENSE00003772050X:154032207-154032557
ENSE00004020471X:154021573-154031450

Protein identifiers

UniProt Accessions

Canonical (Reviewed):

  • P51608 – Methyl-CpG-binding protein 2 (canonical isoform)

Unreviewed (TrEMBL):

  • A0A0D9SEX1
  • A0A0S2Z401
  • A0A140VKC4
  • A0A6Q8PF93
  • A0A6Q8PHQ3
  • A0AAQ5BGE7
  • B5MCB4
  • C9JH89
  • D3YJ43
  • H7BY72

RefSeq Proteins (NP_ accessions)

Reviewed:

  • NP_001104262 (MANE Select, isoform 1)
  • NP_001303266 (isoform 2)
  • NP_001356320 (isoform 3)
  • NP_001356321 (isoform 4)
  • NP_001356322 (isoform 5)
  • NP_001356323 (isoform 6)
  • NP_001373066 (isoform 7)
  • NP_001373067 (isoform 8)
  • NP_001373068 (isoform 9)
  • NP_004983 (isoform 10)
  • NP_010417 (murine, MRX16 variant)

Validated:

  • NP_001075448
  • NP_001315484

Protein Domains and Families

IDNameTypeDatabase
IPR001739Methyl-CpG DNA bindingDomainInterPro
IPR016177DNA-binding domain superfamilyHomologous superfamilyInterPro
IPR017353Methyl-CpG binding protein MeCP2FamilyInterPro
IPR045138Methyl-CpG binding protein MeCP2/MBD4FamilyInterPro
PF01429Methyl-CpG binding domainDomainPfam
SM00391Methyl-CpG DNA bindingDomainSMART
SSF54171DNA-binding domain superfamilySuperfamilySuperfamily (SCOP)
CD01396Methyl-CpG-binding domainDomainCDD/NCBI

Antibody Resources

  • Human Protein Atlas (HPA): Available – ENSG00000169057 has HPA antibody annotations
  • No direct biobtree antibody database matches for MECP2, but HPA provides validated antibodies commonly cited in research

Structure

Experimental Structures

Total PDB entries: 9

PDB IDMethodResolutionDescription
1QK9SOLUTION NMRMeCP2 domain structure binding methylated DNA
3C2IX-RAY DIFFRACTION2.5 ÅMBD domain in complex with methylated DNA sequence (BDNF)
5BT2X-RAY DIFFRACTION2.2 ÅMBD domain (A140V variant) in complex with methylated DNA
6C1YX-RAY DIFFRACTION2.3 ÅMBD domain in complex with methylated DNA
6OGJX-RAY DIFFRACTION1.8 ÅMBD domain in complex with DNA
6OGKX-RAY DIFFRACTION1.65 ÅMBD domain in complex with DNA
6YWWX-RAY DIFFRACTION2.102 ÅFull-length MeCP2 as microsatellite binding protein
8AJRSOLUTION NMRTriple mutant MBD domain
8ALQSOLUTION NMRTriple mutant MBD domain with asymmetrically modified DNA

Summary: 7 X-ray structures, 2 NMR structures

Predicted Structures

AlphaFold Model: P51608

  • Global pLDDT score: 57.25
  • Residues with pLDDT >70 (very high confidence): 9% (0.09 fraction)
  • Sequence length: 3683 residues

Cross-species orthologs

OrganismGene IDSymbol
Mouse17257Mecp2
Rat29386Mecp2
Zebrafish335250mecp2
Fruit flynonenone
Wormnonenone
Yeastnonenone

Based on the data collected, I’ll now compile the summary tables.

Clinical variants & AI predictions

ClinVar Summary

Total variants: ~900 variants in ClinVar (total count shows >900 mapped variants across all pages)

Classification breakdown:

ClassificationCount (approx.)
Pathogenic~120
Likely Pathogenic~80
Benign~150
Likely Benign~300
Uncertain Significance~120
Conflicting/Other~30

Top 30 Pathogenic/Likely Pathogenic variants:

Variant IDHGVS NotationAssociated Condition
11819c.916C>T (p.Arg306Ter)Rett syndrome, Neonatal encephalopathy
11824c.952C>T (p.Arg318Cys)Rett syndrome
11833c.459C>G (p.Tyr153Ter)Rett syndrome
11835c.334C>G (p.Leu112Val)Rett syndrome
11846c.746del (p.Gly249fs)Rett syndrome
143420c.153dup (p.Glu52fs)Rett syndrome
143304c.136_139del (p.Asp46fs)Rett syndrome
143457c.1344_1345del (p.Gln449fs)Rett syndrome
143462c.1360_400del (p.Thr454fs)Rett syndrome
143467c.1393C>T (p.Arg465Ter)Rett syndrome
143487c.1495T>C (p.Ter499Arg)Rett syndrome-related
143491c.182C>A (p.Ser61Ter)Rett syndrome
143492c.182C>G (p.Ser61Ter)Rett syndrome
143497c.230C>G (p.Ser77Ter)Rett syndrome
143499c.237del (p.Ser80fs)Rett syndrome
143500c.239C>G (p.Ser80Ter)Rett syndrome
143502c.251_252insT (p.Ala85fs)Rett syndrome
143503c.251dup (p.Ala85fs)Rett syndrome
143507c.269del (p.Ser90fs)Rett syndrome
143508c.279dup (p.Lys94fs)Rett syndrome
143511c.294_295del (p.Ile99fs)Rett syndrome
143514c.312_313insG (p.Pro105fs)Rett syndrome
143524c.337C>T (p.Pro113Ser)Rett syndrome
143526c.338C>G (p.Pro113Arg)Rett syndrome
143539c.370A>T (p.Lys124Ter)Rett syndrome
143549c.408G>C (p.Leu136Phe)Rett syndrome
143550c.408G>T (p.Leu136Phe)Rett syndrome
143552c.416C>T (p.Pro139Leu)Rett syndrome
143553c.418C>T (p.Gln140Ter)Rett syndrome
11815c.844C>T (p.Arg282Ter)Rett syndrome

AlphaMissense Predictions

Total missense variants with predictions: ~1100+ protein-level variants

Top 30 Likely-Pathogenic (am_class=“likely_pathogenic”) with highest pathogenicity scores:

PositionProtein Variantam_pathogenicity ScoreDelta
X:154030916K304N1.000
X:154030912R306S1.000
X:154030920I303T1.000
X:154030916K304N1.000
X:154030907K307N0.997
X:154030902R309P0.992
X:154030923P302R0.993
X:154030911R306P0.997
X:154030926L301P0.998
X:154030898E310D0.892
X:154030381E483K0.981
X:154030903R309C/G0.980–0.996
X:154030905T308I0.990
X:154030915K305E0.998
X:154030918K304E0.998
X:154030880K307N0.997
X:154030899E310V0.989
X:154030921I303V0.847
X:154030370S486R0.989
X:154030380E483V0.979
X:154030374V485D0.959
X:154030402D476H0.953
X:154030380E483G0.928
X:154030374V485A0.927
X:154030449D460V0.928
X:154030450D460H0.947
X:154030375V485L0.851
X:154030371S486N0.872
X:154030389P480R0.658
X:154030401D476V0.931

Note: Splice prediction databases (SpliceAI, MaxEntScan) returned no direct entries for MECP2; variant annotations can be accessed through transcript-level databases if needed.

Pathways & Gene Ontology

Reactome Pathways

Total: 11 pathways

Pathway IDPathway NameType
R-HSA-8986944Transcriptional Regulation by MECP2Normal
R-HSA-9022534Loss of MECP2 binding ability to 5hmC-DNADisease
R-HSA-9022535Loss of phosphorylation of MECP2 at T308Disease
R-HSA-9022537Loss of MECP2 binding ability to the NCoR/SMRT complexDisease
R-HSA-9022538Loss of MECP2 binding ability to 5mC-DNADisease
R-HSA-9022692Regulation of MECP2 expression and activityNormal
R-HSA-9022699MECP2 regulates neuronal receptors and channelsNormal
R-HSA-9022702MECP2 regulates transcription of neuronal ligandsNormal
R-HSA-9022707MECP2 regulates transcription factorsNormal
R-HSA-9022927MECP2 regulates transcription of genes involved in GABA signalingNormal
R-HSA-9725371Nuclear events stimulated by ALK signaling in cancerDisease

MSigDB Gene Sets

Total: 100 gene sets

Includes GO terms (C5), transcription factor targets (C3:TFT), miRNA targets (C3:MIR), curated pathways (C2:CP), cancer modules (C4), and cytogenetic bands (C1).

Gene Ontology Annotations

Biological Process: 68 terms

GO IDTerm
GO:0000122Negative regulation of transcription by RNA polymerase II
GO:0001662Behavioral fear response
GO:0001666Response to hypoxia
GO:0001964Startle response
GO:0001976Nervous system process involved in regulation of systemic arterial blood pressure
GO:0002087Regulation of respiratory gaseous exchange by nervous system process
GO:0006020Inositol metabolic process
GO:0006357Regulation of transcription by RNA polymerase II
GO:0006541Glutamine metabolic process
GO:0006576Biogenic amine metabolic process
GO:0007219Notch signaling pathway
GO:0007268Chemical synaptic transmission
GO:0007416Synapse assembly
GO:0007420Brain development
GO:0007585Respiratory gaseous exchange by respiratory system
GO:0007612Learning
GO:0007613Memory
GO:0007616Long-term memory
GO:0008104Intracellular protein localization
GO:0008211Glucocorticoid metabolic process

Molecular Function: 14 terms

GO IDTerm
GO:0003676Nucleic acid binding
GO:0003677DNA binding
GO:0003682Chromatin binding
GO:0003714Transcription corepressor activity
GO:0003723RNA binding
GO:0003729mRNA binding
GO:0005515Protein binding
GO:0008327Methyl-CpG binding
GO:0010385Double-stranded methylated DNA binding
GO:0035197siRNA binding
GO:0060090Molecular adaptor activity
GO:0140566Histone reader activity
GO:0140693Molecular condensate scaffold activity
GO:1990841Promoter-specific chromatin binding

Cellular Component: 10 terms

GO IDTerm
GO:0000785Chromatin
GO:0000792Heterochromatin
GO:0005615Extracellular space
GO:0005634Nucleus
GO:0005654Nucleoplasm
GO:0005737Cytoplasm
GO:0005813Centrosome
GO:0005829Cytosol
GO:0045202Synapse
GO:0098794Postsynapse

Protein interactions & networks

Protein-Protein Interactions

Interaction Summary:

  • STRING interactions: ~3,950
  • BioGRID interactions: 1,482
  • IntAct interactions: 239
  • SIGNOR interactions: 49

Top 30 STRING Interactors (by confidence/evidence):

  1. DNMT1 (DNA cytosine-5-methyltransferase 1) — DNA methylation maintenance
  2. SIN3A (Sin3 corepressor complex) — Transcriptional repression partner
  3. HDAC1 (Histone deacetylase 1) — Epigenetic repressor complex
  4. HDAC2 (Histone deacetylase 2) — Epigenetic repressor complex
  5. RCOR1 (REST corepressor 1) — BHC complex, chromatin silencing
  6. NCOR1 (Nuclear receptor corepressor 1) — Transcriptional repression
  7. SUV39H1 (H3K9 histone methyltransferase) — Heterochromatin formation
  8. CREB1 (cAMP-responsive element-binding protein) — Transcription factor
  9. ATRX (Chromatin remodeling helicase) — DNA tandem repeat binding
  10. CDKL5 (Cyclin-dependent kinase-like 5) — MECP2 phosphorylation partner
  11. YBX1 (Y-box binding protein 1) — RNA/DNA binding, m5C recognition
  12. DNMT3A (DNA methyltransferase 3A) — De novo methylation
  13. FMR1 (Fragile X mental retardation protein) — RNA regulation, neuronal function
  14. LBR (Delta-14 sterol reductase) — Nuclear lamina anchoring
  15. DGCR8 (Microprocessor complex subunit) — miRNA biogenesis
  16. BDNF (Brain-derived neurotrophic factor) — Neuronal signaling
  17. DNMT3B (DNA methyltransferase 3B) — De novo methylation
  18. UBE3A (Ubiquitin ligase E3A) — Protein degradation
  19. GAD1 (Glutamate decarboxylase 1) — GABA synthesis
  20. PRMT6 (Protein arginine methyltransferase 6) — H3R2 histone methylation
  21. FOXG1 (Forkhead box G1) — Brain development transcription factor
  22. SIN3B (Sin3 corepressor complex B) — Transcriptional repression
  23. UHRF1 (E3 ubiquitin ligase UHRF1) — DNA methylation/histone binding bridge
  24. H3.2 (Histone H3.2) — Core nucleosome component
  25. TET1 (Methylcytosine dioxygenase) — 5mC to 5hmC conversion
  26. GABRB3 (GABA receptor β3) — GABAergic neurotransmission
  27. H3.3 (Histone H3.3) — Variant histone, active chromatin
  28. H3-5 (Histone H3.3C) — Core nucleosome component
  29. H3.1 (Histone H3.1) — Core nucleosome component
  30. CTCF (Transcriptional repressor) — Chromatin insulator protein

Protein Similarity

ESM2 Embedding Similarity (29 proteins): Top structural similarity includes MBD family proteins, zinc finger proteins, and other methyl-CpG binding domain proteins:

  • Q00566, Q9Z2D6, Q9Z2E1, Q9UBB5, Q95LG8 (MBD family orthologs)
  • P28667, P29536, P35566, P52926, P52927 (Related methyl-binding proteins)
  • Additional zinc finger and chromatin-associated proteins from multiple organisms

Diamond Sequence Similarity (12 proteins):

  • Q9Z2D6, Q9Z2E1, Q9Z2D7, Q9Z2D8 (MeCP2 orthologs across species)
  • Q00566, Q95LG8, Q9UBB5 (MBD protein family members)
  • Q9Z2E2, Q9VGA4, O95243, O95983 (Related methyl-CpG binding proteins)

Key Interaction Networks:

  1. Chromatin Repression Complex: MECP2 → SIN3A/RCOR1 → HDAC1/2 (core corepressor network)
  2. DNA Methylation Axis: MECP2 ↔ DNMT1/3A/3B → UHRF1 (maintenance and establishment)
  3. Histone Modification: MECP2 → SUV39H1/EHMT2/EZH2 → histone variants (H3.1/3.3)
  4. Neuronal Signaling: MECP2 → BDNF/FMR1/CDKL5 → GABAergic signaling (GAD1/GABRB3)
  5. Chromatin Remodeling: MECP2 → ATRX/SMARCA4/BRG1 (DNA accessibility regulation)

Transcription factor regulatory data

MECP2 is a transcription corepressor. GO annotations confirm transcription corepressor activity (GO:0003714) with DNA binding and chromatin binding functions. MECP2 primarily represses transcription through methyl-CpG binding and recruitment of corepressor complexes.

Downstream targets

Total: 40+ targets identified in SIGNOR database

TOP 30 targets with regulation type and evidence:

  1. BDNF — represses (transcriptional regulation, experimentally validated, score 0.47)
  2. ESR1 — represses (transcriptional regulation, score 0.39)
  3. GRIN2B — represses (transcriptional regulation, experimentally validated, score 0.35)
  4. MEF2C — represses (transcriptional regulation, experimentally validated, score 0.35)
  5. CRH — represses (transcriptional regulation, experimentally validated, score 0.47)
  6. NOTCH1 — represses (transcriptional regulation, experimentally validated, score 0.29)
  7. GRIA2 — represses (transcriptional regulation, experimentally validated, score 0.32)
  8. DLX5 — represses (transcriptional regulation, score 0.38)
  9. RELN — represses (transcriptional regulation, experimentally validated, score 0.38)
  10. GAD1 — represses (transcriptional regulation, experimentally validated, score 0.37)
  11. FKBP5 — represses (transcriptional regulation, experimentally validated, score 0.31)
  12. RBFOX1 — represses (transcriptional regulation, experimentally validated, score 0.28)
  13. SGK1 — represses (transcriptional regulation, experimentally validated, score 0.29)
  14. DLL1 — represses (transcriptional regulation, experimentally validated, score 0.20)
  15. PTPN1 — represses (transcriptional regulation, experimentally validated, score 0.20)
  16. AMPA — represses (transcriptional regulation, experimentally validated, score 0.33)
  17. MGMT — represses (transcriptional regulation, score 0.31)
  18. IGFBP3 — represses (transcriptional regulation, score 0.25)
  19. CCND1 — represses (transcriptional regulation, score 0.27)
  20. ALOX5 — represses (transcriptional regulation, score 0.20)
  21. ABCB1 — represses (transcriptional regulation, score 0.20)
  22. TFF1 — represses (transcriptional regulation, score 0.20)
  23. CREB1 — activates (post-transcriptional, experimentally validated, score 0.53)
  24. YBX1 — activates activity (binding, experimentally validated, score 0.51)
  25. DNMT1 — activates activity (binding, experimentally validated, score 0.53)
  26. TET1 — activates activity (binding, experimentally validated, score 0.43)
  27. SST — activates (post-transcriptional, experimentally validated, score 0.30)
  28. GPRIN1 — activates (post-transcriptional, experimentally validated, score 0.30)
  29. GAMT — activates (post-transcriptional, experimentally validated, score 0.28)
  30. OPRK1 — activates (post-transcriptional, experimentally validated, score 0.27)

DNA binding motifs

No JASPAR motifs found. MECP2 does not have characterized DNA binding motifs in JASPAR database. MECP2 recognizes methylated CpG dinucleotides (GO:0008327, GO:0010385) rather than sequence-specific DNA motifs, binding through its methyl-CpG binding domain (MBD).

Upstream regulators

Transcription factors that regulate MECP2:

  1. MEF2C — activates expression (transcriptional regulation, score 0.35)
  2. miR-132 — represses (post-transcriptional regulation, score 0.40)

Post-translational modifiers (with strong evidence):

  1. HIPK2 — activates activity (phosphorylation, experimentally validated, score 0.48)
  2. CDKL5 — modulates (phosphorylation, experimentally validated, score 0.60)
  3. CHUK — activates activity (phosphorylation, experimentally validated, score 0.20)

Drug & pharmacology data

MECP2 as a drug target: Pre-clinical stage with no approved or clinical-stage drugs

Targeting molecules in ChEMBL: 6 total compounds, all in pre-clinical development (Phase 0)

  • CHEMBL1371175 (SID7972424) | Phase 0
  • CHEMBL3687963 (US9034574, XVII) | Phase 0
  • CHEMBL3687966 (US9034574, XXI) | Phase 0
  • CHEMBL3687968 (US9034574, XXIII) | Phase 0
  • CHEMBL489770 (N-(4-(pyridin-2-yl)thiazol-2-yl)pyridin-2-amine, GNF-Pf-4773) | Phase 0
  • CHEMBL520851 (1-(5-phenyl-2,3-dihydro-1,3,4-thiadiazol-2-yl)naphthalen-2-ol, US9034574, XXII) | Phase 0

Clinical trials: Zero clinical trials found targeting MECP2.

Pharmacogenomics: MECP2 is designated a VIP (Very Important Pharmacogene) in PharmGKB with variant annotations. Four drugs have pharmacogenomic associations with MECP2 variants (affecting drug response/toxicity, not targeting the protein):

  • Fluorouracil (197 clinical annotations, 1031 variant annotations)
  • Trofinetide (0 clinical annotations, 0 variant annotations) — developmental MECP2-linked agonist
  • Cisplatin (123 clinical annotations, 541 variant annotations)
  • Mitoxantrone (5 clinical annotations, 24 variant annotations)

Note: No CPIC (Clinical Pharmacogenetics Implementation Consortium) dosing guidelines exist for MECP2.

Expression profiles

Tissue Expression (Bgee)

MECP2 shows ubiquitous expression across 299 conditions with average score of 83.27 (0–100 scale). Top 30 tissues by expression score:

RankTissueScoreQuality
1Paraflocculus97.92Gold
2Brodmann area 1097.01Gold
3Sural nerve96.30Gold
4Frontal pole95.89Gold
5Colonic epithelium95.84Gold
6Middle frontal gyrus95.66Gold
7Cerebellar vermis95.09Gold
8Calcaneal tendon94.44Gold
9Postcentral gyrus92.41Gold
10Tendon91.59Gold
11Parietal lobe91.22Gold
12Blood91.07Gold
13Corpus callosum90.35Gold
14Stomach mucosa90.32Gold
15Bone marrow90.24Gold
16Adrenal tissue90.22Gold
17Granulocytes90.17Gold
18Gastrocnemius89.95Gold
19Leg muscle89.87Gold
20Hindlimb muscle89.82Gold
21Monocytes89.49Gold
22Olfactory nasal mucosa89.38Gold
23Right lung89.22Gold
24Mononuclear leukocytes89.00Gold
25Leukocytes88.98Gold
26Cerebellum88.88Gold
27Parotid gland88.87Silver
28Left ovary88.84Gold
29Tibial artery88.73Gold
30Popliteal artery88.73Gold

Tissue-enriched patterns: CNS-predominant (multiple brain regions, cerebellum), nervous tissue (peripheral nerves), and hematopoietic compartments (blood, bone marrow, granulocytes, monocytes).

Cell Type Expression (Bgee)

Identified cell types include:

  • Hematopoietic: bone marrow cells (90.24), granulocytes (90.17), monocytes (89.49), mononuclear leukocytes (89.00), leukocytes (88.98)
  • Reproductive: secondary oocytes, oocytes, stromal cells of endometrium
  • Respiratory: bronchial epithelial cells

Single-cell Expression (SCXA)

Datasets: 3 studies spanning 27,645 total cells with 280 distinct clusters

DatasetCell Type/ConditionCell Count
Tabula Sapiens (Smart-seq2)Multi-tissue survey27,051
E-MTAB-7381AIRE+ dendritic cells (tonsillitis)384
E-MTAB-7606CD8+ T cells (influenza-specific, baseline/acute/memory)210

Expression metric: Max mean expression 587.84 TPM; average 8.82 TPM across clusters. Gene marked as cluster-defining (marker) in all 3 experiments.

Disease associations

Mendelian / Monogenic Diseases

DiseaseGene IDDisease IDInheritanceEvidence
Rett syndromeMECP2OMIM:312750 / ORPHANET:778 / MONDO:0010726X-linked dominantDefinitive
Severe neonatal-onset encephalopathy with microcephalyMECP2OMIM:300673 / ORPHANET:209370 / MONDO:0010397X-linked dominantDefinitive
Syndromic X-linked intellectual disability Lubs typeMECP2OMIM:300260 / MONDO:0010283X-linked recessiveDefinitive
Chromosome Xq28 duplication syndromeMECP2OMIM:300815 / ORPHANET:1762X-linked dominantDefinitive
X-linked intellectual disability-psychosis-macroorchidism syndromeMECP2ORPHANET:3077 / MONDO:0010235X-linked dominantSupportive
Atypical Rett syndromeMECP2ORPHANET:3095 / MONDO:0017746Autosomal dominantSupportive
Non-syndromic X-linked intellectual disabilityMECP2ORPHANET:777 / MONDO:0019181X-linked dominantSupportive

Phenotype Associations (Top 30 HPO Terms)

HPO IDPhenotypeHPO IDPhenotype
HP:0001249Intellectual disabilityHP:0001298Encephalopathy
HP:0001250SeizureHP:0001250Seizure
HP:0001252HypotoniaHP:0001332Dystonia
HP:0001263Global developmental delayHP:0001336Myoclonus
HP:0000252MicrocephalyHP:0001337Tremor
HP:0001257SpasticityHP:0001417X-linked inheritance
HP:0000717AutismHP:0001508Failure to thrive
HP:0001288Gait disturbanceHP:0000729Autistic behavior
HP:0001344Absent speechHP:0000733Motor stereotypy
HP:0000750Delayed speech and language developmentHP:0001251Ataxia
HP:0001256Mild intellectual disabilityHP:0001258Spastic paraplegia
HP:0002015DysphagiaHP:0000718Aggressive behavior
HP:0000737IrritabilityHP:0001347Hyperreflexia
HP:0000739AnxietyHP:0001266Choreoathetosis
HP:0000713AgitationHP:0001300Parkinsonism

Complex Disease / GWAS Associations

Trait/DiseaseEffect Size (p-value)MECP2 Role
Systemic lupus erythematosus2.0 × 10⁻¹⁵Significant association
Non-albumin protein levels5.0 × 10⁻⁹Genetic variant association

Structured Data Sources

Generated with Claude Haiku 4.5 + BioBTree MCP, drawing on data BioBTree aggregates from 51 biological databases. Every identifier and figure traces to a reproducible API call (listed below).

Further analyze this answer or run your own queries with BioBTree MCP.

Datasets: alphafold, alphamissense, antibody, bgee, biogrid_interaction, ccds, cdd, chembl_molecule, chembl_target, cl, clinical_trials, clinvar, collectri, diamond_similarity, ensembl, entrez, esm2_similarity, exon, expressionatlas, gencc, go, gwas, hgnc, hpa, hpo, intact, interpro, jaspar, maxentscan, mim, mondo, msigdb, omim, orphanet, ortholog, pdb, pfam, pharmgkb, pharmgkb_gene, reactome, refseq, scxa, scxa_expression, signor, smart, spliceai, string, string_interaction, supfam, transcript, uniprot
Generated: 2026-05-25 — For the latest data, query BioBTree directly via MCP or API.
View API calls (142)