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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
81 Results for "
Gene mutation
" in MedChemExpress (MCE) Product Catalog:
Products (81)
81 Results for "Gene mutation" in MCE Product Catalog:
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Cat. No.: HY-W015854
CAS No.: 62-50-0
Synonyms: DSP-5336
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Enzomenib (DSP-5336) is an orally active Menin inhibitor (IC50=1.4 nM, Kd=6.0 nM). Enzomenib disrupts the interaction between Menin and KMT2A/MLL fusion proteins, specifically inhibits the expression of leukemia driver genes such as HOX/MEIS1, and upregulates ITGAM. Enzomenib effectively induces cell differentiation, inhibits tumor cell proliferation, and suppresses primitive cell colony formation. Enzomenib reduces disease burden and prolongs survival, but causes adverse reactions including differentiation syndrome and QTc interval prolongation. Enzomenib is used for research on relapsed/refractory acute myeloid leukemia, acute lymphoblastic leukemia, and other hematologic malignancies with mixed lineage leukemia (MLL) rearrangements or NPM1 mutations .
Cat. No.: HY-16662
CAS No.: 75629-57-1
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Oncrasin-1 is an RNA polymerase inhibitor. Oncrasin-1 suppresses the phosphorylation of the largest subunit of RNA polymerase II and the expression of intronless reporter genes in sensitive cells. Oncrasin-1 effectively kills various human lung cancer cells with K-Ras mutations. Oncrasin-1 leads to coaggregation of PKCι and splicing factors into megaspliceosomes. Oncrasin-1 induces malfunction in the RNA processing machinery. Oncrasin-1 is an anti-cancer agent and can therefore be studied in research for lung cancer .
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TASIN-1 is a selective inhibitor of truncated APC TR (adenomatous polyposis coli gene) that exerts cytotoxic effects by inhibiting cholesterol biosynthesis. TASIN-1 specifically targets colorectal cancer (CRC) cells carrying APC truncated mutations, while having no significant toxicity to wild-type APC cells. TASIN-1 exerts cytotoxic effects by targeting Emopamil binding protein (EBP) to inhibit cholesterol biosynthesis, triggering endoplasmic reticulum (ER) stress, reactive oxygen species (ROS) generation, and JNK-mediated apoptosis, and inhibiting Akt survival signaling. TASIN-1 can be used to prevent and intervene in APC mutant colorectal cancer .
Cat. No.: HY-P99114
CAS No.: 2256084-03-2
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H3B-5942 is a selective, irreversible and orally active estrogen receptor covalent antagonist, inactivates both wild-type and mutant ERα by targeting Cys530, with Kis of 1 nM and 0.41 nM, respectively. H3B-5942 reduces ERα target gene GREB1, shows potent antitumor activity both in multiple cell lines or animals bearing ERα WT or ERα mutations .
Cat. No.: HY-P99047
CAS No.: 1318075-13-6
Synonyms: AB 0024; GS 6624
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Cat. No.: HY-108845
CAS No.: 191588-94-0
Synonyms: TNK-tPA
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Tenecteplase (TNK-tPA) is a modified tissue plasminogen activator. Tenecteplase is a recombinant human tissue plasminogen activator (rt-PA) that has been bioengineered to produce mutations in three gene loci. Tenecteplase (TNK-tPA) can be used in the study of acute ischemic stroke .
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Dihydralazine sulfate is an antihypertensive hydrazine derivative and also a low-potency genotoxic agent. Dihydralazine sulfate is a direct-acting mutagen with a mixed gene mutation mechanism, which induces DNA fragmentation in the lung, kidney and spleen of mice, and induces sister chromatid exchange in mouse bone marrow cells. Dihydralazine sulfate specifically kills DNA repair-deficient bacteria. Dihydralazine sulfate is a vasodilator and antihypertensive agent that reduces systemic vascular resistance, increases cardiac output and heart rate, thereby lowering blood pressure. Dihydralazine sulfate can be used in research related to hypertension and severe early-onset preeclampsia .
Cat. No.: HY-147412
CAS No.: 2642382-41-8
Synonyms: QR-421a
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Ultevursen (QR-421a) is a splice-modulating antisense oligonucleotide targeting exon 13 of the USH2A gene, which restores the functional expression of Usherin protein by inducing exon skipping. Ultevursen binds to USH2A pre-mRNA and modulates the splicing process to specifically skip exon 13 carrying the pathogenic mutation c.2299delG, generating an in-frame transcript and a truncated yet functionally normal protein. Ultevursen exhibits concentration-dependent exon skipping activity in human cells and retinal organoid models, and restores Usherin expression and retinal function in zebrafish and gene-edited mouse models. Ultevursen can be used for related research on type 2 Usher syndrome and non-syndromic retinitis pigmentosa .
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Synonyms: ARO-AAT sodium
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Fazirsiran sodium is a second-generation RNAi agent. Fazirsiran sodium consistes of a cholesterol-conjugated RNAi trigger (chol-RNAi) to selectively degrade Alpha1-antitrypsin (AAT) mRNA by RNAi and a melittin-derived peptide conjugated to N-acetylgalactosamine (NAG) formulated as the excipient EX1 to promote endosomal escape of the chol-RNAi in hepatocytes . Fazirsiran sodium can be used in the study of Alpha-1 Antitrypsin Deficiency (AATD) liver disease. AATD is caused by mutations in the alpha-1 antitrypsin (SERPINA1) gene.
Cat. No.: HY-164552
CAS No.: 920915-26-0
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ZNU-IMB-Z15 (Compound Z15) is an antagonist of the androgen receptor (AR) and also a selective degrader of AR and ARV7. ZNU-IMB-Z15 can directly bind to the ligand-binding domain (LBD) and activation function-1 region of AR, and promote AR degradation through the proteasome pathway. ZNU-IMB-Z15 effectively inhibits the transcriptional activity of AR, AR mutants, and AR splice variants (ARVs), downregulating the mRNA and protein levels of AR downstream target genes, thereby overcoming the resistance to second-generation antiandrogen drugs induced by AR LBD mutations, AR amplification, and ARVs in castration-resistant prostate cancer (CRPC). ZNU-IMB-Z15 can inhibit the proliferation of AR-positive CRPC cell lines and induce their apoptosis, demonstrating anticancer activity both in vivo and in vitro .
Cat. No.: HY-167710
CAS No.: 941185-83-7
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CRCD2 is a small molecule NT5C2 nucleotidase inhibitor with enhanced 6-mercaptopurine cytotoxic activity. CRCD2 can effectively reverse 6-mercaptopurine resistance caused by mutations in the NT5C2 gene or non-genetic activation mechanisms. CRCD2 combined with 6-mercaptopurine can enhance its cytotoxic activity in NT5C2 wild-type leukemia, showing its potential in the treatment of acute lymphoblastic leukemia .
Cat. No.: HY-132604
CAS No.: 2175009-08-0
Synonyms: ARO-AAT
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Fazirsiran (ARO-AAT) is a second-generation RNAi agent. Fazirsiran consistes of a cholesterol-conjugated RNAi trigger (chol-RNAi) to selectively reduce Alpha1-antitrypsin (AAT) synthesis and a melittin-derived peptide conjugated to N-acetylgalactosamine (NAG) formulated as the excipient EX1 to promote endosomal escape of the chol-RNAi in hepatocytes . Fazirsiran can be used in the study of Alpha-1 Antitrypsin Deficiency (AATD) liver disease. Alpha-1 antitrypsin deficiency (AATD) is caused by mutations in the alpha-1 antitrypsin (SERPINA1) gene.
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RNA, (P-thio)(Gm-Gm-Um-Gm-Gm-Am-Um-Cm-Am-Cm-Gm-Am-Gm-Um-Um-Cm-Am)
Cat. No.: HY-153496
CAS No.: 2227173-68-2
Synonyms: QR 110
Cat. No.: HY-174387
CAS No.: 2941104-53-4
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KIN-8741 is a highly selective Type IIb c-Met inhibitor. KIN-8741 has broad activity against c-Met kinase mutations. KIN-8741 shows antitumor activity in MET gene amplified and exon 14 deleted non-small cell lung cancer models. KIN-8741 can be used in the research of c-Met driven cancers, especially advanced tumors carrying MET exon 14 jump mutations, acquired drug resistance mutations, etc .
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Lithium citrate is a lithium supplement that reduces excess brain N-acetylaspartate (NAA) in Canavan disease. Canavan disease is an autosomal recessive leukodystrophy caused by mutations in the aspartate acylase (ASPA) gene, which catalyzes the hydrolysis of N-acetylaspartate (NAA) to acetate and aspartate .
Cat. No.: HY-P11355A
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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
