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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
13 Results for "
inherited diseases
" in MedChemExpress (MCE) Product Catalog:
Products (13)
13 Results for "inherited diseases" in MCE Product Catalog:
2
2 Cited Publications
Cat. No.: HY-161111
CAS No.: 3056394-59-0
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KVS0001 is a selective SMG1 inhibitor. KVS0001 elevates the expression of transcripts and proteins resulting from truncating mutations. KVS0001 increased the presentation of immune-targetable HLA class I-associated peptides from nonsense-mediated decay (NMD)-downregulated proteins on the surface of cancer cells. KVS0001 exerts anti-tumor properties and can be studied in research for NMD-related diseases, including cancer and inherited diseases .
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Human Gut Microbiota Metabolites Ketones, Aldehydes, Acids Metabolic Disease Endogenous metabolite Disease Research Fields
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Synonyms: IkT-148009
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Risvodetinib (IkT-148009) is an orally active, selective and brain-penetrant protein tyrosine kinase inhibitor, displaying excellent target efficacy against c-Abl1, c-Abl2/Arg with IC50 values of 33 nM, 14 nM, respectively. Risvodetinib suppresses c-Abl activation and substantially protects dopaminergic neurons from degeneration in mouse models of both inherited and sporadic Parkinson’s disease (PD), which is promising for research in the field of PD .
Cat. No.: HY-N16119
CAS No.: 53950-58-6
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Cat. No.: HY-113377
CAS No.: 28305-26-2
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Cat. No.: HY-W012734R
CAS No.: 3105-95-1
Synonyms: H-HoPro-OH (Standard)
Cat. No.: HY-W141374
CAS No.: 108883-90-5
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CB096 is an r(G4C2) exp RNA binder with EC50 values of 19 μM, 20 μM and 33 μM. CB096 selectively interacts with the 5′CGG/3′GGC 1×1 GG internal loop motif of folded r(G4C2) exp RNA, alters motif dynamics and closed base pairs, and rescues disease-related pathways. CB096 can be used for research on inherited amyotrophic lateral sclerosis/frontotemporal dementia (c9ALS/FTD) .
Cat. No.: HY-150245
CAS No.: 2839311-21-4
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mHTT-IN-1 (Example 1) is a potent mutant huntingtin (mHTT) inhibitor. mHTT is toxic and a major cause of the inherited autosomal dominant neurodegenerative disorder, Huntington's disease (HD). mHTT-IN-1 conducts the reduction of mHTT with an EC50 value of 46 nM .
Cat. No.: HY-175671
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LSD1/HDAC-IN-3 is a inhibitor targeting class I HDAC and LSD1 enzymes. LSD1/HDAC-IN-3 inhibits HDAC1, HDAC2, HDAC3, and LSD1 with IC50 values of 1702 nM, 842 nM, 358 nM, and 1074 nM, respectively. LSD1/HDAC-IN-3 exhibits antioxidant effects in H2O2-stressed ARPE-19 and 661W retinal cells, increasing levels of acetylated and methylated histone H3. LSD1/HDAC-IN-3 enhances photoreceptor survival in the rd10 mouse model of retinitis pigmentosa. LSD1/HDAC-IN-3 can be used for the study of inherited retinal diseases such as retinitis pigmentosa (RP) .
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Cat. No.: HY-169761
CAS No.: 2879251-63-3
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WIZ-IN-1 (Example 2) is a Wiz inhibitor (DC50: 0.36 μM). WIZ-IN-1 can be used for research of inherited blood disorders (e.g., hemoglobinopathies, e.g., beta- hemoglobinopathies), such as sickle cell disease and beta-thalassemia .
Cat. No.: HY-117051
CAS No.: 1046490-67-8
STA-2842 is an inhibitor of heat shock protein HSP90 with potential to inhibit autosomal dominant polycystic kidney disease (ADPKD). ADPKD is caused by inherited mutations in the PKD1 or PKD2 genes that abnormally activate multiple signaling proteins and pathways that regulate cell proliferation. STA-2842 can significantly reduce initial renal cyst formation and kidney growth in mice, and slow disease progression in mice with existing cysts.
Cat. No.: HY-163818
CAS No.: 2839552-69-9
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WIZ degrader 2 (Compound 142) is a molecular glue degrader for widely interspaced zinc (WIZ) finger motifs with an AC50 of 0.011 μM. WIZ degrader 2 induces the expression of fetal hemoglobin (HbF) with an EC50 of 0.038 μM. WIZ degrader 2 can be used in research related to hereditary blood disorders, such as sickle cell disease (SCD) and thalassemia .
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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
