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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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PEG2000-C-DMG, a pegylated lipid, can be used for the preparation of Onpattro. Onpattro, a hepatically directed investigational RNAi therapeutic agent, harnesses this process to reduce the production of mutant and wild-type transthyretin by targeting the 3′ untranslated region of transthyretin mRNA .
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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
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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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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 .
DMTAP is a cationic lipid that can be used for delivery of DNA, RNAi and drugs .
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 .
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.
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-153484A
CAS No.: 849758-52-7
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Bevasiranib sodium is a siRNA that targets and silences VEGF-A. Bevasiranib sodium activates the RNA-induced silencing complex to degrade VEGF-A mRNA, mediates TLR3-related inhibition of choroidal neovascularization, and triggers RNA interference-mediated gene silencing through endogenous eukaryotic RNAi mechanisms. After intravitreal injection, Bevasiranib sodium distributes to various ocular tissues and resists degradation by intraocular nucleases, reducing choroidal neovascularization area and alleviating vascular leakage. Bevasiranib sodium can be used in research related to macular degeneration .
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-W570886
CAS No.: 163759-97-5
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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 .
Cat. No.: HY-132604
CAS No.: 2175009-08-0
Synonyms: ARO-AAT
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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-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
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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-153484
CAS No.: 959961-96-7
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Research Areas:
Bevasiranib is a siRNA that targets and silences VEGF-A. Bevasiranib activates the RNA-induced silencing complex to degrade VEGF-A mRNA, mediates TLR3-related inhibition of choroidal neovascularization, and triggers RNA interference-mediated gene silencing through endogenous eukaryotic RNAi mechanisms. After intravitreal injection, Bevasiranib distributes to various ocular tissues and resists degradation by intraocular nucleases, reducing choroidal neovascularization area and alleviating vascular leakage. Bevasiranib can be used in research related to macular degeneration .
Cat. No.: HY-P10802
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Cat. No.: HY-158305
CAS No.: 2764770-62-7
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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
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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 .
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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
