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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
16 Results for "
pre-mRNA splicing modulator
" in MedChemExpress (MCE) Product Catalog:
Products (16)
16 Results for "pre-mRNA splicing modulator" in MCE Product Catalog:
7
7 Cited Publications
Cat. No.: HY-N2360
CAS No.: 19202-36-9
Category:
Plant Flavonoids Phenols Polyphenols Selaginella tamariscina (P. Beauv.) Spring Extract Disease Research Fields Biflavones Cancer Selaginellaceae Extract
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Synonyms: PTC518
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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 .
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RNA splicing modulator 3 is an RNA splicing modulator. RNA splicing modulator 3 modulates nucleic acid splicing, including pre-mRNA splicing .
Cat. No.: HY-170935
SRSF1-IN-1 is a SRSF1 inhibitor. SRSF1-IN-1 inhibits SRSF1 expression, thereby modulating the splicing of Bcl-x pre-mRNA. SRSF1-IN-1 inhibits the proliferation of various cancer cells. SRSF1-IN-1 induces apoptosis in gastric cancer cells, reduces Bcl-xl expression, and upregulates cleaved PARP and caspase 3. SRSF1-IN-1 induces autophagy and promotes cell death. SRSF1-IN-1 exhibits anti-tumor activity in a mouse gastric cancer xenograft model. SRSF1-IN-1 can be used for the research of various cancers including liver cancer, gastric cancer, breast cancer, colon cancer, glioma, and melanoma .
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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SMN-C2, an analog of RG-7916, is a selective modulator of SMN2 gene splicing that acts by binding SMN2 pre-mRNA, thereby increasing far upstream element binding protein 1 (FUBP1) and KH-spliced RNA binding Protein affinity regulator protein (KHSRP) to the SMN2 pre-mRNA complex. SMN-C2 can be used in spinal muscular atrophy (SMA) research .
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-143792
CAS No.: 2254502-89-9
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HTT-D3 is an orally active, blood-brain barrier penetrant splicing modulator of huntingtin (HTT). HTT-D3 promotes the inclusion of a pseudo-exon containing a premature termination codon into HTT pre-mRNA, triggers nonsense-mediated mRNA degradation and reduces HTT protein levels. HTT-D3 induces dose-dependent, comparable reductions in mutant HTT protein in both the brain and peripheral tissues of transgenic mouse models. HTT-D3 can be used for the research of Huntington's disease .
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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-N2360R
CAS No.: 19202-36-9
Cat. No.: HY-W278073
CAS No.: 430443-35-9
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RNA splicing modulator 6 (Compound c7) is a RNA splicing modulator. RNA splicing modulator 6 induces changes in pre-mRNA splicing. RNA splicing modulator 6 causes accumulation of unspliced RNA in the Hs-Hsh155 strain. RNA splicing modulator 6 reduces proliferation of chronic myeloid leukemia cells harboring the cancer-associated SRSF2 P95H mutation .
Cat. No.: HY-189713
CAS No.: 2650573-17-2
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PTC-553 is a microtubule-associated protein tau (MAPT) mRNA splicing modulator. PTC-553 promotes exon 10 exclusion by modulating MAPT pre-mRNA splicing, corrects the 4R/3R tau isoform imbalance, thereby reducing the pathological accumulation of 4R tau protein and alleviating neurotoxicity. PTC-553 can be used for research on frontotemporal dementia and tauopathies .
Cat. No.: HY-189714
CAS No.: 3025238-38-1
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PTC-775 is a MAPT pre-mRNA splicing modulator. PTC-775 promotes MAPT exon exclusion, reduces longer tau isoforms and increases shorter isoforms. PTC-775 reduces the accumulation of total tau and hyperphosphorylated tau species (including oligomeric and insoluble forms). PTC-775 protects neurons from glutamate-induced death. PTC-775 can be used for research on frontotemporal dementia .
Cat. No.: HY-147412D
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FAM labled Ultevursen sodiumis a FAM labled Ultevursen sodium (HY-147412A). Ultevursen sodium (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 sodium 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 sodium 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 sodium can be used for related research on type 2 Usher syndrome and non-syndromic retinitis pigmentosa .
Cat. No.: HY-147412E
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Cy3 labled Ultevursen sodium is a Cy3 labled Ultevursen sodium (HY-147412A). Ultevursen sodium (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 sodium 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 sodium 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 sodium can be used for related research on type 2 Usher syndrome and non-syndromic retinitis pigmentosa .
Cat. No.: HY-189715
CAS No.: 3025238-91-6
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PTC-003 is an orally active and blood-brain barrier-penetrant MAPT pre-mRNA splicing modulator. PTC-003 promotes skipping of exon 10 of the MAPT gene, shifting the protein isoform balance from toxic 4R-tau toward 3R-tau. PTC-003 reduces the accumulation of hyperphosphorylated tau, oligomeric tau, and insoluble tau protein. PTC-003 protects neurons from glutamate-induced neuronal stress injury, and in humanized MAPT gene-replaced N279K mice, it corrects the 3R/4R-tau isoform ratio in brain tissue and reduces the level of pathogenic pTau T231. PTC-003 can be used in research related to neurodegenerative diseases .
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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
