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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
40 Results for "
skipping
" in MedChemExpress (MCE) Product Catalog:
Products (40)
40 Results for "skipping" in MCE Product Catalog:
Synonyms: SRP-4053
Target:
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Golodirsen (SRP-4053) is an antisense oligonucleotide of the phophorodiamidate morpholino oligomer (PMO). Golodirsen restores the reading frame of the Duchenne muscular dystrophy (DMD) gene by modifying the splicing process of the pre-mRNA, skipping exon 53. Golodirsen can restore the expression of the anti-myostatin protein. Golodirsen can be used for the research of duchenne muscular dystrophy (DMD) .
Cat. No.: HY-132586
CAS No.: 2055732-84-6
Synonyms: NS-065/NCNP-01
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Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen has the potential for Duchenne muscular dystrophy (DMD) research .
Synonyms: Kyndrisa; GSK2402968A; PRO051
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Drisapersen (Kyndrisa) is a 2 '-O-methyl phosphorothioate RNA antisense oligonucleotide that induces exon 51 skipping. Drisapersen induces skipping of exon 51 during Dystrophin pre-mRNA splicing, allowing the synthesis of partially functional Dystrophin. Drisapersen can be used in research related to Duchenne muscular dystrophy .
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Kinesore is an inhibitor of the KLC2-SKIP Interaction.
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RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G), 5'-[P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylphosphonamidate], sodium salt
Cat. No.: HY-108753A
Purity: 91.16%
Synonyms: AVI 4658 sodium
Cat. No.: HY-132611A
Purity: 92.00%
Synonyms: SRP-4053 sodium
Target:
Research Areas:
Golodirsen (SRP-4053) sodium is an antisense oligonucleotide of the phophorodiamidate morpholino oligomer (PMO). Golodirsen sodium restores the reading frame of the Duchenne muscular dystrophy (DMD) gene by modifying the splicing process of the pre-mRNA, skipping exon 53. Golodirsen sodium can restore the expression of the anti-myostatin protein. Golodirsen sodium can be used for the research of duchenne muscular dystrophy (DMD) .
Cat. No.: HY-147412
CAS No.: 2642382-41-8
Synonyms: QR-421a
Research Areas:
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 .
Cat. No.: HY-132586A
Purity: 95.25%
Synonyms: NS-065/NCNP-01 sodium
Research Areas:
Viltolarsen (NS-065/NCNP-01) sodium is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen sodium binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen sodium has the potential for Duchenne muscular dystrophy (DMD) research .
Target:
Research Areas:
CD33 splicing modulator 1 (Compound 1) is a CD33 splicing modulator. CD33/Siglec 3 is a myeloid lineage cell surface receptor that is known to regulate microglia activity. CD33 splicing modulator 1 increases exon 2 skipping in cellular mRNA pools. CD33 splicing modulator 1 has the potential for the research of neurodegenerative disease including Alzheimer's disease .
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RNA, (P-thio)(Um-Cm-Am-Am-Gm-Gm-Am-Am-Gm-Am-Um-Gm-Gm-Cm-Am-Um-Um-Um-Cm-Um), sodium salt
Synonyms: Kyndrisa sodium; GSK2402968A sodium; PRO051 sodium
Cat. No.: HY-132584A
Purity: 99.07%
Synonyms: SRP-4045 sodium
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Casimersen (SRP-4045) sodium is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer subclass. Casimersen sodium binds to exon 45 of dystrophin pre-mRNA, restores the open-reading frame (by skipping exon 45) resulting in the production of an internally truncated but functional dystrophin protein. Casimersen sodium can be used for the research of Duchenne muscular dystrophy (DMD) .
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RNA, (P-thio)[2′-O-(2-methoxyethyl)](A-G-m5C-m5U-m5U-m5C-G-G-A-G-A-A-A-m5U-m5U-m5U-A-A-A-m5U-m5C), sodium salt
Cat. No.: HY-147412A
Synonyms: QR-421a sodium
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RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-Am5U-m5U-m5U-C-m5U-A-[2'a-(N2-acetyl-L-arginyl-L-arginyl-L-arginyl-Larginyl-L-arginyl-L-arginylglycyl)]G), 5'-[P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,Ndimethylphosphonamidate], sodium salt
Cat. No.: HY-132585A
Synonyms: SRP-5051 sodium
Cat. No.: HY-158143
CAS No.: 2918814-97-6
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AZD3470 is an orally active MTA-cooperative PRMT5 inhibitor, selective for MTAP-deficient tumors. AZD3470 induces cell cycle G2/M phase alterations, DNA damage, apoptosis, and symmetric dimethylarginine reduction. AZD3470 alters alternative splicing, increases skipped exon events in DNA repair and cell cycle pathways, and inhibits cancer cell proliferation and tumor growth. AZD3470 can be used for the research of non-small cell lung cancer and MTAP-deleted solid tumors .
Cat. No.: HY-177658
CAS No.: 1782108-31-9
Synonyms: DS-5141b
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Renadirsen is an antisense oligonucleotide that induces robust Exon 45 skipping for Dystrophin in vivo.
Cat. No.: HY-177659
CAS No.: 1196915-71-5
Target:
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Rimigorsen is an antisense oligonucleotide that induces skipping of exon 44 of the pre-mRNA encoding dystrophin in a Duchenne muscular dystrophy (DMD)
Cat. No.: HY-144399A
CAS No.: 2762547-06-6
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CD33 splicing modulator 1 (Compound 1) hydrochloride is a CD33 splicing modulator. CD33/Siglec 3 is a myeloid cell surface receptor known to regulate microglial activity. CD33 splicing modulator 1 hydrochloride increases exon 2 skipping in the cellular mRNA pool and can be used to study neurodegenerative diseases including Alzheimer's disease .
Cat. No.: HY-E70751
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Research Areas:
Mesenchymal-to-epithelial transition (MET) is a receptor tyrosine kinase for hepatocyte growth factor (HGF). MET overactivation is strongly associated with angiogenesis, cellular motility, growth, and invasion. Aberrant MET signaling can drive tumorigenesis in several cancer types through various molecular mechanisms, including MET amplification, MET exon 14 skipping mutation, MET overexpression, and MET fusions. MET G1163R is a mutant of MET. MET G1163R Recombinant Human Active Protein Kinase is a recombinant MET G1163R protein that can be used to study MET G1163R-related functions .
Cat. No.: HY-E70758
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Research Areas:
Mesenchymal-to-epithelial transition (MET) is a receptor tyrosine kinase for hepatocyte growth factor (HGF). MET overactivation is strongly associated with angiogenesis, cellular motility, growth, and invasion. Aberrant MET signaling can drive tumorigenesis in several cancer types through various molecular mechanisms, including MET amplification, MET exon 14 skipping mutation, MET overexpression, and MET fusions. MET Y1235D is a mutant of MET. MET Y1235D Recombinant Human Active Protein Kinase is a recombinant MET Y1235D protein that can be used to study MET Y1235D-related functions .
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RNA, ((2'-deoxy-2'-fluoro)U-[P(S)]-sp-(2'-deoxy-2'-fluoro)C-[P(S)]-sp-(2'-deoxy-2'-fluoro)A-[P(S)]-sp-(2'-deoxy-2'-fluoro)A-[P(S)]-sp-(2'-deoxy-2'-fluoro)G-[P(S)]-sp-(2'-deoxy-2'-fluoro)G-[P(S)]-sp-Am-(2'-deoxy-2'-fluoro)A-[P(S)]-sp-Gm-Am-[P(S)]-sp-(2'-deoxy-2'-fluoro)U-[P(S)]-sp-Gm-Gm-(2'-deoxy-2'-fluoro)C-[P(S)]-sp-(2'-deoxy-2'-fluoro)A-[P(S)]-sp-(2'-deoxy-2'-fluoro)U-[P(S)]-sp-(2'-deoxy-2'-fluoro)U-[P(S)]-sp-(2'-deoxy-2'-fluoro)U-[P(S)]-sp-(2'-deoxy-2'-fluoro)C-[P(S)]-sp-(2'-deoxy-2'-fluoro)U), sodium salt
Cat. No.: HY-132592A
CAS No.: 2142024-01-7
Synonyms: WVE-210201 sodium
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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
