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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
146 Results for "
exon
" in MedChemExpress (MCE) Product Catalog:
Products (146)
146 Results for "exon" in MCE Product Catalog:
Synonyms: PTC518
Target:
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Votoplam (PTC518) is a blood-brain barrier-permeable HTT pre-mRNA inhibitor with an IC50 ≤ 0.1 μM. Votoplam modulates the splicing process of HTT pre-mRNA to include a pseudo-exon carrying a premature translation termination codon, thereby triggering HTT mRNA degradation. Votoplam is applicable to research related to Huntington's disease .
BAY 2476568 is a potent and mutant-selective inhibitor targeting EGFR exon20 insertion variants. BAY 2476568 potently inhibits the kinase activity of EGFR exon20 insertion mutants (insASV, insSVD, insNPG) with IC50 values of 0.09 nM, 0.21 nM, and 0.11 nM, respectively. BAY 2476568 inhibits EGFR (Y1068) phosphorylation and reduces the phosphorylation of ERK1/2 and Akt (S473) in Ba/F3 cells expressing EGFR exon20 insertion mutants (insASV, insSVD). BAY 2476568 can be used for the study of non-small cell lung cancer (NSCLC) driven by EGFR exon20 insertion mutations .
Cat. No.: HY-108753A
Purity: 91.16%
Synonyms: AVI 4658 sodium
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Eteplirsen (AVI 4658) sodium is a phosphorylated diamine morpholino oligonucleotide that targets exon 51 of the human Duchenne muscular dystrophy (DMD) gene. Eteplirsen sodium induces exon 51 skipping, causing it to be skipped during splicing, thereby restoring the translation reading frame and producing a shortened functional dystrophin. Eteplirsen sodium can be used in research on Duchenne muscular dystrophy .
Cat. No.: HY-132611A
Purity: 92.00%
Synonyms: SRP-4053 sodium
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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
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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 .
Cat. No.: HY-153402
CAS No.: 1380600-06-5
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Activated EG3 Tail can be used in the synthesis of exon jumping oligomer conjugates .
Cat. No.: HY-132586A
Purity: 95.25%
Synonyms: NS-065/NCNP-01 sodium
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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 .
Cat. No.: HY-177360
CAS No.: 3067915-73-2
Synonyms: RNA splicing modulator-4
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Ruxuplam (RNA splicing modulator-4) is an RNA splicing modulator. Ruxuplam controls the inclusion or exclusion of specific exons in precursor mRNA (pre-mRNA) by regulating alternative splicing events, thereby altering the coding sequence and function of mature mRNA. Ruxuplam shows promise for research into neurodegenerative diseases (such as Huntington's disease) and cancers .
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TG693 is an orally active inhibitor of CLK1. TG693 regulates the mutated exon 31 of the dystrophin gene in vivo. TG693 is used in Duchenne muscular dystrophy (DMD) research .
Cat. No.: HY-164307
CAS No.: 2769954-39-2
Synonyms: BLU 451; LNG-451
Pebezertinib (BLU 451) is an orally active inhibitor for EGFR. Pebezertinib exhibits the ability to penetrate the central nervous system (CNS). Pebezertinib can be used for research about non-small cell lung cancer carrying EGFR exon 20 insertion .
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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-174215
CAS No.: 3063511-09-8
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TAU-IN-3 (Compound 2) is an orally active TAU inhibitor. TAU-IN-3 inhibits the expression of MAPT exon 10 DDPAC mutant gene in HeLa cells (IC50: 0.6 µM). TAU-IN-3 reduces the 4R/3R MAPT mRNA ratio in HeLa cells transfected with WT or DDPAC minigenes. TAU-IN-3 inhibits the insertion of endogenous MAPT exon 10 and the production of 4R tau protein in cells. TAU-IN-3 modulates tau splicing in htau mice and improves the associated behavioral phenotypes. TAU-IN-3 can be used to study neurodegenerative diseases .
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Activated DPG Subunit can be used in the synthesis of exon jumping oligomer conjugates. The oligomer conjugates complement selected target sites in the human anti-muscular atrophy protein gene and induce exon 51 jumping. It can be used for research of muscular dystrophy .
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Activated T Subunit can be used in the synthesis of exon jumping oligomer conjugates. The oligomer conjugates complement selected target sites in the human anti-muscular atrophy protein gene and induce exon 51 jumping. Activated T subunit can be used for research of muscular dystrophy .
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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
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) .
Cat. No.: HY-132585A
Synonyms: SRP-5051 sodium
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Vesleteplirsen sodium (SRP-5051 sodium) is a peptide-conjugated morpholino oligonucleotide (PPMO) that mediates exon jumping molecular effects. Vesleteplirsen sodium targets exon 51 of the DMD gene pre-mRNA, remodeling the transcript splicing process, restoring the protein translation read frame, and generating internally truncated pseudodystrophy proteins. Vesleteplirsen sodium can be used for research related to Duchenne muscular dystrophy .
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 .
Cat. No.: HY-123766
CAS No.: 2068806-31-3
EGFR-IN-99 (compound 1a) is a potent EGFR and HER2 Exon 20 insertion mutant inhibitor. EGFR-IN-99 has excellent antiproliferative activity against DFCI127 cells, with an EC50 of 11.5 nM. EGFR-IN-99 can be used for the research of non-small cell lung cancer (NSCLC) .
Cat. No.: HY-W753281
CAS No.: 3936-02-5
Dexamethasone metasulfobenzoate sodium is a SARS-CoV-2 exonuclease (ExoN) inhibitor that binds to the catalytic site of ExoN .
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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
