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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
64 Results for "
pre-mRNA splicing
" in MedChemExpress (MCE) Product Catalog:
Products (64)
64 Results for "pre-mRNA splicing" in MCE Product Catalog:
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-136951
CAS No.: 347397-83-5
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cp028 is a potent pre-mRNA splicing inhibitor. cp028 inhibits splicing in HeLa nuclear extract with an IC50 value of 54 µM .
Cat. No.: HY-169794
CAS No.: 37571-96-3
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N-Palmitoyl-phenylalanine is an N-acyl amide that contains the long-chain saturated fatty acid Palmitic acid (HY-N0830) conjugated via an amide bond to the essential amino acid L-Phenylalanine (HY-N0215). N-Palmitoyl-phenylalanine inhibits pre-mRNA splicing in HeLa cell nuclear lysates (IC50 ＞ 400 μM) but has no splicing activity in intact HeLa cells .
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)(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-21286
CAS No.: 63264-29-9
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N2-Isobutyryl-2'-O-methylguanosine is a nucleic acid synthesis intermediate (e.g., used in antisense oligonucleotides, mRNA modification), for example, it is a key monomer for the synthesis of 2'-O-methyl oligoribonucleotides. N2-Isobutyryl-2'-O-methylguanosine enables the final product to form stable double strands with complementary RNA and is not easily degraded by nucleases. N2-Isobutyryl-2'-O-methylguanosine is mainly used in molecular biology research, and can be used to prepare RNA hybridization probes or participate in related biochemical research such as pre-mRNA splicing mechanisms .
Cat. No.: HY-139103
CAS No.: 1426953-24-3
Category:
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Cat. No.: HY-132585A
Synonyms: SRP-5051 sodium
Research Areas:
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 .
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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-109101R
CAS No.: 1825352-65-5
Synonyms: RG7916 (Standard); RO7034067 (Standard)
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Risdiplam (Standard) is the analytical standard of Risdiplam. This product is intended for research and analytical applications. Risdiplam (RG7916) is an orally administered, centrally and peripherally distributed SMN2 pre-mRNA splicing modifier that increases survival motor neuron (SMN) protein levels .
Cat. No.: HY-N2360R
CAS No.: 19202-36-9
Cat. No.: HY-13650R
CAS No.: 165668-41-7
Synonyms: E 7070 (Standard)
Research Areas:
Indisulam (Standard) (E 7070 (Standard)) is the analytical standard of Indisulam (HY-13650). This product is intended for research and analytical applications. Indisulam (E 7070) is a carbonic anhydrase inhibitor and RBM39 molecular glue degrader, with Ki values of 31, 15, and 24 nM against human carbonic anhydrase I, II, and IX, respectively, and a Ki value of 65 nM against bovine carbonic anhydrase IV. Indisulam promotes the recruitment of RBM39 to the CUL4-DCAF15 E3 ubiquitin ligase complex, triggering polyubiquitination and proteasomal degradation of RBM39. Indisulam induces aberrant pre-mRNA splicing, G1 cell cycle arrest, and cell death in cancer cells. As an anticancer agent, Indisulam drives tumor regression and growth inhibition in xenograft models. Indisulam can be used in research related to solid tumors, hematologic and lymphoid malignancies, colorectal cancer, and non-small cell lung cancer .
Cat. No.: HY-139102
CAS No.: 1426953-23-2
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Thailanstatin B is a potent pre-mRNA splicing inhibitor (IC50=6.18 μM). Thailanstatin B shows antiproliferative activity .
Cat. No.: HY-150237
Purity: 92.99%
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FITC-labeled Drisapersen (sodium) is Drisapersen labeled with FITC. Drisapersen, a antisense oligonucleotide, induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthesis of partially functional dystrophin in Duchenne muscular dystrophy (DMD) patients with amenable mutations.
Cat. No.: HY-109101AS1
Cat. No.: HY-109101AS2
Cat. No.: HY-122637
CAS No.: 2071636-70-7
Sudemycin K is a selective splicing inhibitor targeting SF3B1. Sudemycin K blocks U2 snRNP recognition of branch point sequences in pre-mRNA, inducing tumor-specific splicing deregulation and apoptosis. Sudemycin K is promising for research of SF3B1-mutated malignancies such as chronic lymphocytic leukemia (CLL) and myelodysplastic syndromes (MDS) .
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 .
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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
