Details of the Drug Therapeutic Target (DTT)

General Information of Drug Therapeutic Target (DTT) (ID: TTTAU9R)

• DTT Name: Methyl cpg binding protein 2 (MECP2)
• Synonyms: MethylCpGbinding protein 2; Methyl-CpG-binding protein 2; MeCp2 protein; MeCp2; MeCp-2 protein
• Gene Name: MECP2
• DTT Type: Literature-reported target; [1]
• UniProt ID: MECP2_HUMAN
• TTD ID: T59686
• Sequence:
  MVAGMLGLREEKSEDQDLQGLKDKPLKFKKVKKDKKEEKEGKHEPVQPSAHHSAEPAEAG
  KAETSEGSGSAPAVPEASASPKQRRSIIRDRGPMYDDPTLPEGWTRKLKQRKSGRSAGKY
  DVYLINPQGKAFRSKVELIAYFEKVGDTSLDPNDFDFTVTGRGSPSRREQKPPKKPKSPK
  APGTGRGRGRPKGSGTTRPKAATSEGVQVKRVLEKSPGKLLVKMPFQTSPGGKAEGGGAT
  TSTQVMVIKRPGRKRKAEADPQAIPKKRGRKPGSVVAAAAAEAKKKAVKESSIRSVQETV
  LPIKKRKTRETVSIEVKEVVKPLLVSTLGEKSGKGLKTCKSPGRKSKESSPKGRSSSASS
  PPKKEHHHHHHHSESPKAPVPLLPPLPPPPPEPESSEDPTSPPEPQDLSSSVCKEEKMPR
  GGSLESDGCPKEPAKTQPAVATAATAAEKYKHRGEGERKDIVSSSMPRPNREEPVDSRTP
  VTERVS
• Function: It can bind specifically to a single methyl-CpG pair. It is not influenced by sequences flanking the methyl-CpGs. Mediates transcriptional repression through interaction with histone deacetylase and the corepressor SIN3A. Binds both 5-methylcytosine (5mC) and 5-hydroxymethylcytosine (5hmC)-containing DNA, with a preference for 5-methylcytosine (5mC). Chromosomal protein that binds to methylated DNA.
• Reactome Pathway:
  Loss of MECP2 binding ability to 5hmC-DNA (R-HSA-9022534)
  Loss of phosphorylation of MECP2 at T308 (R-HSA-9022535)
  Loss of MECP2 binding ability to the NCoR/SMRT complex (R-HSA-9022537)
  Loss of MECP2 binding ability to 5mC-DNA (R-HSA-9022538)
  Regulation of MECP2 expression and activity (R-HSA-9022692)
  MECP2 regulates neuronal receptors and channels (R-HSA-9022699)
  MECP2 regulates transcription of neuronal ligands (R-HSA-9022702)
  MECP2 regulates transcription factors (R-HSA-9022707)
  MECP2 regulates transcription of genes involved in GABA signaling (R-HSA-9022927)
  Nuclear events stimulated by ALK signaling in cancer (R-HSA-9725371)
  Transcriptional Regulation by MECP2 (R-HSA-8986944)

Molecular Interaction Atlas (MIA) of This DTT

1 Clinical Trial Drug(s) Targeting This DTT

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
AVXS-201	DM8Z4U3	Rett syndrome	LD90.4	Phase 1	[2]

References

• [1] MECP2, a multi-talented modulator of chromatin architecture. Brief Funct Genomics. 2016 Nov;15(6):420-431.
• [2] Clinical pipeline report, company report or official report of Novartis