Scientific Reviews
Product Guides
Blogs
Topics
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
50 Results for "
Splicing activity
" in MedChemExpress (MCE) Product Catalog:
Products (50)
50 Results for "Splicing activity" in MCE Product Catalog:
Cat. No.: HY-147412D
Target:
Research Areas:
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
Target:
Research Areas:
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-114461A
CAS No.: 1228183-92-3
Target:
Research Areas:
(16R,17S)-FD-895, a similar compound to FD-895 (HY-114461), is a potent in vivo splice regulatory agent. (16R,17S)-FD-895 retains the similar anti-proliferative activity as FD-895. (16R,17S)-FD-895 can be used for research on colon cancer .
Cat. No.: HY-177972
Research Areas:
Zotadirsen is the core component of the AOC drug Delpacibart zotadirsen (AOC1044) (HY-177564), consisting of a phosphorodiamidate morpholino oligonucleotide (PMO) targeting exon 44 of the dystrophin gene and an SMCC linker (HY-42360). Upon conjugation with the anti-TfR1 antibody Delpacibart (HY-P990051), Zotadirsen exerts splicing regulatory activity and mediates exon skipping. Zotadirsen can be used in studies related to AOC synthesis .
Cat. No.: HY-171808
CAS No.: 2798907-16-9
Target:
Research Areas:
ITRI-90 is an orally effective androgen receptor (AR) PROTAC degrader. ITRI-90 effectively degrades full-length AR (AR-FL) and its splice variant AR-V7 proteins via the ubiquitin-proteasome system, thereby inhibiting AR transcriptional activity and the expression of its target genes, and further inducing tumor cell apoptosis. ITRI-90 can be used in research related to castration-resistant prostate cancer and Enzalutamide (HY-70002)-resistant prostate cancer .
Cat. No.: HY-W1058476
CAS No.: 1443367-20-1
Research Areas:
GPR35 agonist 6 is a potent and selective agonist targeting GPR35, with an EC50 of 12.1 nM against human GPR35. GPR35 agonist 6 activates the human splice variant GPR35b with comparable efficacy, showing an EC50 of 22.1 nM. GPR35 agonist 6 also exhibits weak GPR55 antagonistic activity, with an IC50 of 21.7 μM, and its agonistic activity towards GPR35 is significantly stronger than its antagonistic activity towards GPR55. GPR35 agonist 6 initiates GPR35 downstream signaling by inducing β-arrestin recruitment. GPR35 agonist 6 is used to investigate GPR35-mediated diseases, such as gastrointestinal lesions and gastric cancer .
Cat. No.: HY-187172
CAS No.: 2060801-74-1
Target:
Research Areas:
BDF34019555 is a blood-brain barrier-permeable binder targeting the conserved region (amino acid residues 320-340) of TDP-43, with a Kd of 10.4 μM for full-length TDP-43 and 7.8 μM for the CR polypeptide, respectively. BDF34019555 binds to the conserved α-helical region of TDP-43 in a Trp334-dependent manner, inhibits liquid-liquid phase separation, aggregation and mitochondrial localization of TDP-43, restores mitochondrial function, reduces cytoplasmic and insoluble TDP-43 levels, without affecting the splicing activity of TDP-43. BDF34019555 exerts neuroprotective effects, alleviates motor neuron loss and improves motor neuron function. BDF34019555 can be used in studies related to amyotrophic lateral sclerosis .
Cat. No.: HY-P11698
CAS No.: 476667-31-9
Research Areas:
Cat. No.: HY-L248
858 compounds
The RNA-targeted bioactive compound library is a high-quality collection of small molecules specifically designed and curated to target RNA structures and functions. It is widely applied in cutting-edge drug discovery and life science research. Unlike traditional strategies that focus on protein targets, RNA-targeted compounds can directly modulate various functional RNA molecules by influencing their splicing, translation, stability, or structural conformation, thereby enabling precise intervention in key biological processes. In the field of drug development, these compounds provide a novel approach to addressing previously “undruggable” targets and have demonstrated significant potential in areas such as oncology, antiviral therapies, and neurodegenerative diseases. For example, by targeting disease-associated RNA structural domains or regulating the aberrant expression of non-coding RNAs, these compounds can effectively inhibit disease progression or restore normal cellular function. In mechanistic studies, RNA-targeted compounds serve as valuable chemical biology tools to elucidate the roles of RNA in gene expression regulation, cellular signaling pathways, and disease development.
The MCE RNA-targeted bioactive compound library contains 858 compounds, sourced from databases such as TargetRX Atlas and R-BIND. The library features excellent structural diversity and biological activity, making it suitable for high-throughput screening (HTS), target validation, phenotypic screening, and lead compound discovery. It represents a valuable resource for RNA-related research and innovative drug development.
Cat. No.: HY-123996
CAS No.: 20041-64-9
Research Areas:
3-Ethoxy-5,6-dibromosalicylaldehyde is an IRE1/ERN1 inhibitor, with an IC50 of 0.12 μM, a Ki of 71-88 nM, and a Kd of 100 nM against the ribonuclease activity of hIRE1α, as well as an IC50 of 4.8 μM against yeast Ire1. It shows selectivity toward IRE1 ribonuclease. 3-Ethoxy-5,6-dibromosalicylaldehyde blocks the IRE1/ERN1-mediated unfolded protein response (UPR) signaling pathway, including XBP-1 mRNA splicing, induction of XBP1 target genes, and activation of MAPK8/9/10, but does not alter the phosphorylation level of IRE1α or the PERK/ATF6 pathway. 3-Ethoxy-5,6-dibromosalicylaldehyde inhibits chikungunya virus replication, induces growth arrest, apoptosis and clonogenic inhibition in pancreatic cancer cells, reduces FB1 (Fumonisin B1) (HY-N6719)-induced autophagy and cell death, regulates PGG-induced senescence and apoptosis, and alleviates Sorafenib (HY-10201)-induced vacuolization and damage in hepatic stellate cells. 3-Ethoxy-5,6-dibromosalicylaldehyde can be used in research related to chikungunya virus infection, pancreatic cancer, FB1-induced nephrotoxicity, liver cancer, breast cancer, lung cancer and liver fibrosis .
-
-
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
