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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
21 Results for "
SMN2 splicing
" in MedChemExpress (MCE) Product Catalog:
Products (21)
21 Results for "SMN2 splicing" in MCE Product Catalog:
18
Target:
Research Areas:
Branaplam (LMI070; NVS-SM1) is a highly potent, selective and orally active survival motor neuron-2 (SMN2) splicing modulator with an EC50 of 20 nM for SMN. Branaplam inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. Branaplam elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model .
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18 Publications Verification
Cat. No.: HY-19620A
CAS No.: 1562338-39-9
Synonyms: LMI070 hydrochloride; NVS-SM1 hydrochloride
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Research Areas:
Branaplam (LMI070; NVS-SM1) hydrochloride is a highly potent, selective and orally active survival motor neuron-2 (SMN2) splicing modulator with an EC50 of 20 nM for SMN. Branaplam hydrochloride inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. Branaplam hydrochloride elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model .
11
11 Cited Publications
Synonyms: RG7916; RO7034067
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Risdiplam (RG7916) is an orally administered, centrally and peripherally distributed SMN2 pre-mRNA splicing modifier that increases survival motor neuron (SMN) protein levels .
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7 Cited Publications
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Research Areas:
Nusinersen is an antisense oligonucleotide active molecule. Nusinersen modifies the pre-messenger RNA splicing of the SMN2 gene, thereby promoting the production of full-length SMN protein. Nusinersen improves spinal muscular atrophy .
7
7 Cited Publications
Cat. No.: HY-112980A
Purity: 98.91%
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Research Areas:
Nusinersen sodium is an antisense oligonucleotide active molecule. Nusinersen sodium modifies the pre-messenger RNA splicing of the SMN2 gene, thereby promoting the production of full-length SMN protein. Nusinersen sodium improves spinal muscular atrophy .
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RG7800 is a SMN2 splicing modifier. RG7800 has the potential for spinal muscular atrophy treatment.
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RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein; RG7800 hydrochloride has the potential to treat spinal muscular atrophy.
1
1 Cited Publications
Cat. No.: HY-109101S
Purity: 98.88%
Synonyms: PK4C9
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Homocarbonyltopsentin (PK4C9) is a small-molecule TSL2-binding compound, binds to pentaloop conformations of TSL2 and promotes a shift to triloop conformations that display enhanced SMN2 exon 7 (E7) splicing with EC50 value of 16 μM .
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SMN-C3 is an orally active SMN2 splicing modulator and has the potential to treat spinal muscular atrophy (SMA).
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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 .
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NVS-SM2 is a potent, orally active and brain-penetrant SMN2 splicing enhancer with an EC50 of 2 nM for SMN. NVS-SM2 enhances U1-pre-mRNA association. NVS-SM2 promotes exon 7 inclusion and restores normal survival motor neuron (SMN) protein expression. NVS-SM2 can be used for spinal muscular atrophy (SMA) research .
Cat. No.: HY-121473
CAS No.: 60504-57-6
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Aklavin is a structural analog of Aclacinomycin A (HY-N2306) produced by Streptomyces strain A 1165. Aklavin possesses Z-DNA-inducing and stabilizing activities, along with antibiotic, anti-phage and broad-spectrum antimicrobial activities. Aklavin inhibits the proliferation of various viruses (such as influenza virus and poliovirus) and interferes with their nucleoprotein synthesis, while also exhibiting inhibitory effects on staphylococci, mycobacteria and specific fungi. Aklavin blocks phage-induced bacterial lysis by regulating host-parasite interactions. Aklavin shows specific toxicity to fertilized eggs and mice, and does not alter the splicing of the SMN2 gene .
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-109101AS1
Cat. No.: HY-109101AS2
Cat. No.: HY-133895
CAS No.: 1449592-94-2
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SMN-C5 is an inducer of SMN2 exon 7 inclusion. SMN-C5 binds to the SMN2 exon 7 5′-splice site/U1 snRNA helix (Kd = 28 μM) and positively allosterically modulates SMN2 splicing correction. SMN-C5 binds to SMN2 exon 7 ESE2 and partially displaces hnRNP G, enhances the affinity of U1 snRNP for the weak splice site, binds to a bulged adenine in the major groove and forms hydrogen bonds, while not affecting the interaction of Tra2-β1 with ESE2 RNA. SMN-C5 can be used for research on spinal muscular atrophy .
Cat. No.: HY-189605
CAS No.: 2407724-51-8
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SMN modulator-1 is an SMN2 mRNA splicing modulator. SMN modulator-1 increases the expression of full-length SMN (FL-SMN) mRNA by promoting the inclusion of exon 7 in the SMN2 gene. SMN modulator-1 can be used in research related to spinal muscular atrophy (SMA) .
Cat. No.: HY-101792R
CAS No.: 1449598-06-4
Synonyms: RO6885247 (Standard)
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Research Areas:
RG7800 (Standard) (RO6885247 (Standard)) is the analytical standard of RG7800 (HY-101792). This product is intended for research and analytical applications. RG7800 is a SMN2 splicing modifier. RG7800 has the potential for spinal muscular atrophy treatment.
Cat. No.: HY-153269
CAS No.: 2248691-80-5
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9-Methyl-SMN-C5 is a SMN2 pre-mRNA splicing modifier that corrects exon 7 splicing. 9-Methyl-SMN-C5 binds to ESE2 and the 5'-splice site of exon 7, and stabilizes the RNP complex. 9-Methyl-SMN-C5 induces chemical shift perturbations at the 5'-splice site of the exon 7-U1 snRNA duplex. 9-Methyl-SMN-C5 partially blocks the binding of hnRNP G to ESE2. 9-Methyl-SMN-C5 can be used for research on spinal muscular atrophy (SMA) .
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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
