Target Information
| Target General Information | Top | |||||
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| Target ID |
T64645
(Former ID: TTDI02180)
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| Target Name |
N-sulphoglucosamine sulphohydrolase (SGSH)
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| Synonyms |
Sulphamidase; Sulfoglucosamine sulfamidase; HSS
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| Gene Name |
SGSH
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| Target Type |
Clinical trial target
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[1] | ||||
| Disease | [+] 1 Target-related Diseases | + | ||||
| 1 | Lysosomal disease [ICD-11: 5C56] | |||||
| Function |
Catalyzes a step in lysosomal heparan sulfate degradation.
Click to Show/Hide
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| BioChemical Class |
Sulfur-nitrogen hydrolase
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| UniProt ID | ||||||
| EC Number |
EC 3.10.1.1
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| Sequence |
MSCPVPACCALLLVLGLCRARPRNALLLLADDGGFESGAYNNSAIATPHLDALARRSLLF
RNAFTSVSSCSPSRASLLTGLPQHQNGMYGLHQDVHHFNSFDKVRSLPLLLSQAGVRTGI IGKKHVGPETVYPFDFAYTEENGSVLQVGRNITRIKLLVRKFLQTQDDRPFFLYVAFHDP HRCGHSQPQYGTFCEKFGNGESGMGRIPDWTPQAYDPLDVLVPYFVPNTPAARADLAAQY TTVGRMDQGVGLVLQELRDAGVLNDTLVIFTSDNGIPFPSGRTNLYWPGTAEPLLVSSPE HPKRWGQVSEAYVSLLDLTPTILDWFSIPYPSYAIFGSKTIHLTGRSLLPALEAEPLWAT VFGSQSHHEVTMSYPMRSVQHRHFRLVHNLNFKMPFPIDQDFYVSPTFQDLLNRTTAGQP TGWYKDLRHYYYRARWELYDRSRDPHETQNLATDPRFAQLLEMLRDQLAKWQWETHDPWV CAPDGVLEEKLSPQCQPLHNEL Click to Show/Hide
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| 3D Structure | Click to Show 3D Structure of This Target | PDB | ||||
| Drugs and Modes of Action | Top | |||||
|---|---|---|---|---|---|---|
| Clinical Trial Drug(s) | [+] 4 Clinical Trial Drugs | + | ||||
| 1 | LYS-SAF302 | Drug Info | Phase 2/3 | Mucopolysaccharidosis | [2] | |
| 2 | HGT-1410 | Drug Info | Phase 2 | Sanfilippo syndrome | [3] | |
| 3 | ABO-102 | Drug Info | Phase 1/2 | Mucopolysaccharidosis | [4] | |
| 4 | SOBI003 | Drug Info | Phase 1/2 | Sanfilippo syndrome | [5] | |
| Mode of Action | [+] 2 Modes of Action | + | ||||
| Replacement | [+] 2 Replacement drugs | + | ||||
| 1 | LYS-SAF302 | Drug Info | [6] | |||
| 2 | SOBI003 | Drug Info | [8] | |||
| Modulator | [+] 1 Modulator drugs | + | ||||
| 1 | HGT-1410 | Drug Info | [1] | |||
| Target Affiliated Biological Pathways | Top | |||||
|---|---|---|---|---|---|---|
| KEGG Pathway | [+] 3 KEGG Pathways | + | ||||
| 1 | Glycosaminoglycan degradation | |||||
| 2 | Metabolic pathways | |||||
| 3 | Lysosome | |||||
| Reactome | [+] 1 Reactome Pathways | + | ||||
| 1 | HS-GAG degradation | |||||
| WikiPathways | [+] 1 WikiPathways | + | ||||
| 1 | Glycosaminoglycan metabolism | |||||
| References | Top | |||||
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| REF 1 | ClinicalTrials.gov (NCT01155778) Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients. U.S. National Institutes of Health. | |||||
| REF 2 | ClinicalTrials.gov (NCT03612869) Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) (AAVance). U.S. National Institutes of Health. | |||||
| REF 3 | ClinicalTrials.gov (NCT02350816) Safety and Efficacy Study for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. U.S. National Institutes of Health. | |||||
| REF 4 | ClinicalTrials.gov (NCT04088734) Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease. U.S. National Institutes of Health. | |||||
| REF 5 | ClinicalTrials.gov (NCT03811028) A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients. U.S. National Institutes of Health. | |||||
| REF 6 | Clinical pipeline report, company report or official report of Lysogene. | |||||
| REF 7 | Clinical pipeline report, company report or official report of Abeona Therapeutics. | |||||
| REF 8 | Clinical pipeline report, company report or official report of Swedish Orphan Biovitrum. | |||||
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