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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
26 Results for "
RNAi
" in MedChemExpress (MCE) Product Catalog:
Products (26)
26 Results for "RNAi" in MCE Product Catalog:
4
4 Cited Publications
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Research Areas:
Nedosiran (DCR-PHXC) sodium is an RNA interference (RNAi) targeting lactate dehydrogenase (LDH). Nedosiran sodium represents an impactful potential therapeutic for primary hyperoxaluria (PH) with end-stage renal disease (ESRD). Nedosiran sodium is a GalNAc-dsRNA conjugate .
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1 Cited Publications
Research Areas:
Lumasiran sodium, an investigational RNA interference (RNAi) therapeutic agent, reduces hepatic oxalate production by targeting glycolate oxidase. Lumasiran sodium reduces urinary oxalate excretion, the cause of progressive kidney failure in primary hyperoxaluria type 1 (PH1) .
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Research Areas:
Nedosiran (DCR-PHXC) is an RNA interference (RNAi) targeting lactate dehydrogenase (LDH). Nedosiran represents an impactful potential therapeutic for primary hyperoxaluria (PH) with end-stage renal disease (ESRD). Nedosiran is a GalNAc-dsRNA conjugate .
Cat. No.: HY-145720
CAS No.: 1639264-46-2
Synonyms: ALN-CC5
Research Areas:
Cemdisiran (ALN-CC5) is an N-acetylgalactosamine-conjugated RNAi agent and also a complement component C5 inhibitor. Cemdisiran targets C5 mRNA, cleaves C5 mRNA via the endogenous RNA interference pathway, and inhibits the production of C5 protein in the liver. Cemdisiran exerts a dose-dependent inhibitory effect on total C5 concentrations in cynomolgus monkeys. When used in combination with Pozelimab (HY-P99786) in cynomolgus monkeys, Cemdisiran achieves a more sustained and complete inhibitory effect on complement activity. Cemdisiran can be used in the research of paroxysmal nocturnal hemoglobinuria and other complement-mediated diseases .
Cat. No.: HY-147426
CAS No.: 2437257-11-7
Synonyms: ADS-007; ARO HIF2
Research Areas:
Zifcasiran (ADS-007; ARO HIF2) is an siRNA synthetic double-stranded RNAi trigger. Zifcasiran sodium selectively target hypoxia-inducible factor-2α (HIF2α) interrupting downstream pro-oncogenic signaling in clear cell renal cell carcinoma (ccRCC). Zifcasiran sodium engages the cell's RNAi machinery to target HIF2α (EPAS1) mRNA for degradation, thereby reducing the amount of free HIF2α mRNA available for translation .
Cat. No.: HY-150224
Purity: 97.93%
Research Areas:
GalNAc unconjugated/naked Fitusiran (sodium), an small interfering RNA without GalNAc conjugation, specifically targets antithrombin (AT) messenger RNA to lower production of AT in the liver. Fitusiran increases thrombin generation and has the potential for the research of the hemophilia .
Synonyms: ARO-AAT sodium
Target:
Research Areas:
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-W570886
CAS No.: 163759-97-5
Target:
Research Areas:
2'-O-MOE-U is a nucleic acid modification group (Phosphoramidite) with 3'-exonuclease inhibitory activity. 2'-O-MOE-U also exhibits gene silencing activity and double-stranded oligonucleotide stability. By forming steric interactions with 3'-exonuclease residues, 2'-O-MOE-U anchors the 3'-end of the siRNA guide strand in the hAgo2 PAZ domain, thereby regulating double-stranded thermal stability and enhancing base-pairing specificity. 2'-O-MOE-U does not induce IFNα production, can be incorporated at multiple sites of siRNA to enhance RNAi activity, and produces a synergistic effect with 2'-F modification. 2'-O-MOE-U has been widely used in studies related to breast cancer and other diseases .
IR415 is a potent anti-HBV agent and inhibits HBV replication by blocking the HBx activity. IR415 selectively interacts with HBx (Kd=2 nM) and blocks HBV-mediated RNAi suppression, reverses the inhibitory effect of HBx protein on the activity of the dicer endoribonuclease . HBx: hepatitis B virus X protein.
Cat. No.: HY-145720A
Purity: 96.02%
Synonyms: ALN-CC5 sodium
Research Areas:
Cemdisiran sodium (ALN-CC5 sodium) is an N-acetylgalactosamine-conjugated RNAi agent and also a complement component C5 inhibitor. Cemdisiran sodium targets C5 mRNA, cleaves C5 mRNA via the endogenous RNA interference pathway, and inhibits the production of C5 protein in the liver. Cemdisiran sodium exerts a dose-dependent inhibitory effect on total C5 concentrations in cynomolgus monkeys. When used in combination with Pozelimab (HY-P99786) in cynomolgus monkeys, Cemdisiran sodium achieves a more sustained and complete inhibitory effect on complement activity. Cemdisiran sodium can be used in the research of paroxysmal nocturnal hemoglobinuria and other complement-mediated diseases .
Cat. No.: HY-132604
CAS No.: 2175009-08-0
Synonyms: ARO-AAT
Target:
Research Areas:
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.
Cat. No.: HY-W004287
CAS No.: 1731-88-0
Category:
Target:
Cat. No.: HY-147426A
CAS No.: 2437257-12-8
Synonyms: ADS-007 sodium; ARO HIF2 sodium
Research Areas:
Zifcasiran (ADS-007) sodium is an siRNA synthetic double-stranded RNAi trigger. Zifcasiran sodium selectively target hypoxia-inducible factor-2α (HIF2α) interrupting downstream pro-oncogenic signaling in clear cell renal cell carcinoma (ccRCC). Zifcasiran sodium engages the cell's RNAi machinery to target HIF2α (EPAS1) mRNA for degradation, thereby reducing the amount of free HIF2α mRNA available for translation .
Cat. No.: HY-402873
CAS No.: 2382942-38-1
Target:
Research Areas:
DMTr-2'-O-C16-rC(Ac)-3'-CE-phosphoramidite (Compound 5) is an RNAi agent and an inhibitor of Ataxin-2 (ATXN2). DMTr-2'-O-C16-rC(Ac)-3'-CE-phosphoramidite can be studied in research on ATXN2-associated neurological diseases .
Cat. No.: HY-158305
CAS No.: 2764770-62-7
Target:
Research Areas:
SM51a is a small molecule targeting ligand. SM51a can be linked to the 3' or 5' end of a sense strand or an antisense strand of a DUX4 RNAi agent for promoting it entry into cells .
Cat. No.: HY-158306
CAS No.: 2764770-56-9
Target:
Research Areas:
SM45a is a small molecule targeting ligand. SM45a can be linked to the 3' or 5' end of a sense strand or an antisense strand of a DUX4 RNAi agent for promoting it entry into cells .
Cat. No.: HY-172328
O12-D3-I3 is an ionizable cationic lipid. O12-D3-I3 can be used in the generation of lipid nanoparticles (LNPs) for siRNA delivery in vitro and in vivo. LNPs containing O12-D3-I3 enhances LNP endosomal escape for ferroptosis RNAi therapy of cancer .
Cat. No.: HY-176863
CAS No.: 3094598-90-7
Target:
Research Areas:
Di(5-(methylsulfonyl)-1,3,4-oxadiazol-2-yl)-benzene-amide-PEG4-ester-2,3,5,6-F-Ph (Compound L-1026) is a linker that connects Androgen Receptor (AR) RNAi agent to targeting ligands (such as antibodies). The conjugation can inhibit AR gene expression and reduce AR activity. Di(5-(methylsulfonyl)-1,3,4-oxadiazol-2-yl)-benzene-amide-PEG4-ester-2,3,5,6-F-Ph can be used for spinal and bulbar muscular atrophy (SBMA) research .
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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
