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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
76 Results for "
SMN gem
" in MedChemExpress (MCE) Product Catalog:
Products (76)
76 Results for "SMN gem" in MCE Product Catalog:
16
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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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16 Publications Verification
Cat. No.: HY-19620A
CAS No.: 1562338-39-9
Synonyms: LMI070 hydrochloride; NVS-SM1 hydrochloride
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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 .
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9 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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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 .
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7 Cited Publications
Cat. No.: HY-112980A
Purity: 98.91%
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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.
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4 Cited Publications
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AA147 is a endoplasmic reticulum (ER) proteostasis regulator. AA147 promotes protection against oxidative damage in neuronal cells and prevents endothelial barrier dysfunction by activating ATF6 arm (selectively) of the unfolded protein response (UPR) and the NRF2 oxidative stress response. AA147 can rebalances XBP1s expression in vivo, and also induces survival motor neuron (SMN) expression and spinal motorneuron (MN) protection .
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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).
Cat. No.: HY-W750960
CAS No.: 1377605-22-5
Biotinylated isoxazole is a chemical probe that selectively precipitates proteins containing prion-like low-complexity domains and Cross-β prion-like polymers through recognition of Cross-β structure. Biotinylated isoxazole induces precipitation of proteins containing intrinsically disordered regions through microcrystal formation in solution. Biotinylated isoxazole enables enrichment of Amyloid oligomers for ELISA-based detection. Biotinylated isoxazole decreases Gems and increases misfolded PFN1 aggregates .
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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-143411
CAS No.: 2487526-28-1
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GEM144 is a potent and orally active DNA polymerase α (POLA1) and HDAC 11 dual inhibitor. GEM144 induces acetylation of p53, activation of p21, G1/S cell cycle arrest, and apoptosis. GEM144 has significant antitumor activity in human orthotopic malignant pleural mesothelioma xenografts .
Cat. No.: HY-149906
CAS No.: 153021-75-1
Synonyms: gem91
Trecovirsen (GEM91) is an antiviral agent targeting HIV gag mRNA, which hybridizes with complementary HIV gag mRNA at the initiation site. Trecovirsen induces a reversible, dose-dependent prolongation of activated partial thromboplastin time via its polyanionic property. Trecovirsen is applicable to research related to HIV infection .
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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-149906C
Purity: 98.83%
Synonyms: gem91 sodium
Trecovirsen sodium is a 25-mer antisense phosphorothioate oligonucleotide targeted at the gag site of the HIV gene.
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ML372 inhibits survival motor neuron (SMN) protein ubiquitination, increases SMN protein stability without affecting mRNA expression. ML372 improves spinal muscular atrophy (SMA) in mice. ML372 is brain penetrant and has a reasonable exposure and half-life in vivo .
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 .
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RNA, [2'-O-(2-methylamino)-2-oxoethyl](m5C-sp-A-m5C-sp-m5U-m5U-sp-m5U-sp-m5C-sp-A-sp-m5U-sp-A-sp-A-sp-m5U-sp-G-sp-m5C-sp-m5U-sp-G-sp-G-sp-m5C)
Cat. No.: HY-177661
CAS No.: 2701584-16-7
Synonyms: BIIB115; ION306
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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
