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Targeted therapy! The Capetin Prize winning "Click Chemistry" can be used like this!Targeted therapy! The Nobel Prize winning "Click Chemistry" can be used like this!2025-02-06
18 Results for "
lysosomal storage disorders
" in MedChemExpress (MCE) Product Catalog:
Products (18)
18 Results for "lysosomal storage disorders" in MCE Product Catalog:
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1 Cited Publications
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UGT8-IN-1 is a brain penetrable and orally active inhibitor of ceramide galactosyltransferase enzyme (UGT8). UGT8-IN-1 can be used in the study for lysosomal storage disorders .
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Galactosylceramide (GalC) is a galactosylceramide that has been implicated in many physiological and pathological phenomena. For example, important lysosomal storage disorders such as Gaucher disease (GD) and neurodegenerative diseases are characterized by GlcCer accumulation.
Cat. No.: HY-12733
CAS No.: 1254318-44-9
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AZD5248 is an orally active, selective dipeptidyl peptidase 1 (cathepsin C) inhibitor, with IC50 values of 1 nM and 17 nM against human CatC, 44 nM against human DPP1, and 67 nM against rat DPP1. It exhibits low clearance and high bioavailability in animal models. AZD5248 forms an irreversible covalent bond with the catalytic Cys234 residue of CatC, exerts reversible inhibition via its nitrile moiety, blocks CatC-dependent amyloid formation, and reduces the activation levels of neutrophil serine proteases in bone marrow and blood. AZD5248 reacts with aortic elastin aldehydes to form stable 4-imidazolinones, induces ultrastructural changes in aortic tissue, and has an α-amino acid-based backbone. AZD5248 reduces the severity of acute pancreatitis in mouse models. AZD5248 can be used in research on chronic obstructive pulmonary disease, acute pancreatitis, neurodegenerative diseases, lysosomal storage disorders, acute lung injury, cystic fibrosis, and neutrophil-mediated inflammatory diseases .
Cat. No.: HY-145549
CAS No.: 121999-68-6
Synonyms: N-eicosanoyl-D-erythro-Sphingosylphosphorylcholine; SM(d18:1/20:0)
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C20 Sphingomyelin (d18:1/20:0) (N-eicosanoyl-D-erythro-Sphingosylphosphorylcholine; SM(d18:1/20:0)) is a naturally occurring sphingolipid. C20 Sphingomyelin (d18:1/20:0) levels are upregulated in the hippocampus of streptozotocin (HY-13753)-induced diabetic rats and in human plasma, positively correlating with insulin resistance in obese humans. C20 Sphingomyelin (d18:1/20:0) is also upregulated in the liver of a mouse model of Niemann-Pick C1 disease, a neurodegenerative cholesterol sphingolipid lysosomal storage disorder.
Cat. No.: HY-178202
CAS No.: 2734265-58-6
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5-C-phenethyl-DNJ is a selective α-glucosidase GAA inhibitor, with its Ki value for rhGAA being 0.81 μM. 5-C-phenethyl-DNJ exhibits extremely high selectivity for GANAB, GBA1, and GBA2. 5-C-phenethyl-DNJ can be used for the study of Pompe disease .
Cat. No.: HY-153830S
CAS No.: 2097249-92-6
Synonyms: C16 Lactosylceramide (d18:1/16:0)-d3
Cat. No.: HY-186121
CAS No.: 3118501-69-9
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Glucosylceramide synthase-IN-5 (G3-3) is a glucosylceramide synthase (GCS) inhibitor that can be used for the study of the diseases and disorders associated with GCS activity, such as lysosomal storage disorders .
Cat. No.: HY-186121A
CAS No.: 3118501-71-3
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Glucosylceramide synthase-IN-6 (G3-3B) is a glucosylceramide synthase (GCS) inhibitor that can be used for the study of the diseases and disorders associated with GCS activity, such as lysosomal storage disorders .
Cat. No.: HY-156020
CAS No.: 851185-20-1
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Glucocerebrosidase-IN-2 (compound 12) is a quinazoline analogue and an inhibitor of glucocerebrosidase (GC). Glucocerebrosidase-IN-2 has the potential to improve GC translocation to lysosomes in Gaucher disease patient-derived cells (mostly carrying the N370S mutation). Glucocerebrosidase-IN-2 inhibits the hydrolysis of 4-methylumbelliferone β-D-glucopyranoside (4MU) and fluorescent glycosylceramide (FlourGC) in N370S mutant tissues with an AC50 of 25.29 μM .
Cat. No.: HY-E70305
CAS No.: 84012-69-1
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GlcNAc-1-Phosphotransferase is an α2β2γ2 hexamer with core catalytic α- and β-subunits derived from GNPTAB. GlcNAc-1-Phosphotransferase catalyzes the addition of the il group of N-acetylglucosamine-1-phosphate to the terminal mannose. Mutations in GlcNAc-1-Phosphotransferase cause lysosomal storage disorders such as mucolipidosis .
Cat. No.: HY-186254
CAS No.: 3129652-92-9
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TMEM175 agonist 2 is a TMEM175 agonist with an EC50 of 0.0446 μM in human FLIPR assays and an EC50 of 0.0453 μM in human TMEM175 EP assays. TMEM175 agonist 2 can be used in research related to Parkinson's disease, dementia with Lewy bodies, rapid eye movement sleep behavior disorder, amyotrophic lateral sclerosis, and lysosomal storage diseases .
Cat. No.: HY-186257
CAS No.: 3129652-66-7
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TMEM175 agonist 3 (Compound 123) is a TMEM175 agonist, with an EC50 of 0.044 μM in human TMEM175 FLIPR assays and an EC50 of 0.073 μM in human TMEM175 EP assays. TMEM175 agonist 3 can be used in the research of Parkinson's disease, dementia with Lewy bodies, rapid eye movement sleep behavior disorder, amyotrophic lateral sclerosis, and lysosomal storage diseases .
Cat. No.: HY-186253
CAS No.: 3129653-69-3
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TMEM175 agonist 1 is a TMEM175 agonist, with a human EC50 of 0.378 μM in FLIPR assays and a human EC50 of 0.886 μM in EP analyses. TMEM175 agonist 1 can be used in the research of Parkinson's disease, dementia with Lewy bodies, rapid eye movement sleep behavior disorder, amyotrophic lateral sclerosis, and lysosomal storage diseases .
Cat. No.: HY-186261
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TMEM175 agonist 7 is a TMEM175 agonist with an EC50 of 543 nM. TMEM175 agonist 7 is applicable to the research of neurodegenerative diseases and lysosomal storage disorders .
Cat. No.: HY-186259
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TMEM175 agonist 5 (Compound 252) is a TMEM175 agonist with an EC50 of 421 nM in FLIPR cell assays. TMEM175 agonist 5 can be used for the research of neurodegenerative diseases and lysosomal storage disorders .
Cat. No.: HY-184340
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α-Glucosidase-IN-118 is a non-competitive inhibitor of α-glucosidase with a IC50 of 2.77 μM, and it shows no inhibitory activity against β-glucosidase. α-Glucosidase-IN-118 binds to the entrance of the α-glucosidase active site to block substrate release. α-Glucosidase-IN-118 can be used in the research of diabetes, obesity and lysosomal storage diseases .
Cat. No.: HY-184093
CAS No.: 2648407-86-5
Synonyms: Rexaceractum
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Rexaceract (Rexaceractum) is a heteroaryl compound that binds to β-glucocerebrosidase. Rexaceract maintains the conformational stability of β-glucocerebrosidase and exhibits activating activity. Rexaceract can be used in the research of nervous system-related diseases such as neurodegenerative diseases with Parkinsonism, epilepsy, and schizophrenia .
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Protocols
Targeted therapy! The Capetin Prize winning "Click Chemistry" can be used like this!Targeted therapy! The Nobel Prize winning "Click Chemistry" can be used like this!A suitable model is crucial in drug screening experiments. Organs can mimic the three-dimensional functional structure of internal organs, have similar spatial organization to corresponding organs, maintain some key characteristics, and reproduce some physiological functions. They are widely used for modeling and personalized drug screening physiological functions.2025-02-06
