Details of the Drug Therapeutic Target (DTT)

General Information of Drug Therapeutic Target (DTT) (ID: TT0IUKX)

DTT Name	Alpha-L-iduronidase (IDUA)
Gene Name	IDUA
DTT Type	Clinical trial target	[1]
UniProt ID	IDUA_HUMAN
TTD ID	T98430
3D Structure	Download 2D Sequence (FASTA) Download 3D Structure (PDB) Download
EC Number	EC 3.2.1.76
Sequence	MRPLRPRAALLALLASLLAAPPVAPAEAPHLVHVDAARALWPLRRFWRSTGFCPPLPHSQ ADQYVLSWDQQLNLAYVGAVPHRGIKQVRTHWLLELVTTRGSTGRGLSYNFTHLDGYLDL LRENQLLPGFELMGSASGHFTDFEDKQQVFEWKDLVSSLARRYIGRYGLAHVSKWNFETW NEPDHHDFDNVSMTMQGFLNYYDACSEGLRAASPALRLGGPGDSFHTPPRSPLSWGLLRH CHDGTNFFTGEAGVRLDYISLHRKGARSSISILEQEKVVAQQIRQLFPKFADTPIYNDEA DPLVGWSLPQPWRADVTYAAMVVKVIAQHQNLLLANTTSAFPYALLSNDNAFLSYHPHPF AQRTLTARFQVNNTRPPHVQLLRKPVLTAMGLLALLDEEQLWAEVSQAGTVLDSNHTVGV LASAHRPQGPADAWRAAVLIYASDDTRAHPNRSVAVTLRLRGVPPGPGLVYVTRYLDNGL CSPDGEWRRLGRPVFPTAEQFRRMRAAEDPVAAAPRPLPAGGRLTLRPALRLPSLLLVHV CARPEKPPGQVTRLRALPLTQGQLVLVWSDEHVGSKCLWTYEIQFSQDGKAYTPVSRKPS TFNLFVFSPDTGAVSGSYRVRALDYWARPGPFSDPVPYLEVPVPRGPPSPGNP
Function	extracellular exosome, lysosomal lumen, hydrolase activity, hydrolyzing O-glycosyl compounds, L-iduronidase activity, chondroitin sulfate catabolic process, dermatan sulfate catabolic process, disaccharide metabolic process, glycosaminoglycan catabolic process, heparin catabolic process
KEGG Pathway	Glycosaminoglycan degradation (hsa00531 ) Metabolic pathways (hsa01100 ) Lysosome (hsa04142 )
Reactome Pathway	CS/DS degradation (R-HSA-2024101 ) MPS I - Hurler syndrome (R-HSA-2206302 ) HS-GAG degradation (R-HSA-2024096 )
BioCyc Pathway	MetaCyc:HS05096-MON

Molecular Interaction Atlas (MIA) of This DTT

Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This DTT

2 Clinical Trial Drug(s) Targeting This DTT

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
SB-318	DMA8Z4N	Mucopolysaccharidosis	5C56.3	Phase 1/2	[1]
RGX-111	DMU87HW	Mucopolysaccharidosis	5C56.3	Phase 1	[2]
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The Drug-Metabolizing Enzyme (DME) Role of This DTT

DTT DME Name

Alpha-L-iduronidase (IDUA) DME Info

Gene Name

IDUA

1 Approved Drug(s) Metabolized by This DTT

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
Chondroitin sulfate	DM0N19Y	N. A.	N. A.	Phase 4	[3]
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References

1	ClinicalTrials.gov (NCT02702115) Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I. U.S. National Institutes of Health.
2	Clinical pipeline report, company report or official report of REGENXBIO.
3	MFN human ref: Chondroitin sulfate degradation.