Search Result

Results for "

RNA splicing

" in MedChemExpress (MCE) Product Catalog:

By Targets:	DNA/RNA Synthesis (14) ADC Payload (1) Androgen Receptor (1) Apoptosis (1) CDK (1) Fluorescent Dye (2) Huntingtin (1) iGluR (2) Ras (1)
By Research Areas:	Cancer (7) Infection (1) Inflammation/Immunology (2) Metabolic Disease (1) Neurological Disease (6)
Oligonucleotides:	Antisense Oligonucleotides (4)

Inhibitors & Agonists

Screening Libraries

Peptides

Natural
Products

Oligonucleotides

Targets Recommended: RNA MTase DNA/RNA Synthesis Small Interfering RNA (siRNA) mRNA SF3B1

Cat. No.	Product Name	Target	Research Areas	Chemical Structure

HY-150248

RNA splicing modulator 3	DNA/RNA Synthesis	Others
RNA splicing modulator 3 (compound 236) is a RNA splicing modulator, with an AC₅₀ value of <100 nM .

HY-P5960

PTBP1-RNA-binding inhibitor P6 TFA PTBP1 α3-helix derived peptide P6 TFA	DNA/RNA Synthesis	Others
PTBP1-RNA-binding inhibitor P6 (PTBP1 α3-helix derived peptide P6) TFA is a stapled peptide inhibitor of the splicing factor PTBP1, which inhibits alternative splicing events regulated by PTBP1. PTBP1 binds RNA through its RNA recognition motif .

HY-153736

NSC 194308	DNA/RNA Synthesis	Cancer
NSC 194308, a *U2AF2-RNA* complexes enhancer, increases association of the U2AF1-U2AF2-SF1-splice site RNA complex by binding a site between the U2AF2 RNA recognition motifs (RRM1 and RRM2). NSC 194308 inhibits pre-mRNA splicing by stalling spliceosome assembly at the point where U2AF helps recruit U2 snRNP to the branchpoint. NSC 194308 enhances the binding of pre-mRNA to U2AF2, selectively triggering cell death in leukemia cell lines containing spliceosome mutations .

HY-150247

RNA splicing modulator 2	DNA/RNA Synthesis	Others
RNA splicing modulator 2 (compound 256) is a RNA splicing modulator .

HY-150246

RNA splicing modulator 1	DNA/RNA Synthesis	Others
RNA splicing modulator 1 (compound 233) is a RNA splicing modulator, with an AC₅₀ value of <100 nM .

HY-148384A

UHMCP1 dihydrochloride	DNA/RNA Synthesis	Cancer
UHMCP1 dihydrochloride is a potent UHM domain splicing inhibitor with a K_d value of 79 μM. UHMCP1 dihydrochloride prevents the SF3b155/U2AF ⁶⁵ interaction. UHMCP1 dihydrochloride impacts cell viability and RNA splicing .

HY-148384

UHMCP1	Fluorescent Dye	Cancer
UHMCP1 is a chemical probe of U2AF homology motifs (UHM) with a K_d of 79 μM. UHMCP1 prevents the SF3b155/U2AF65 interaction, impacts RNA splicing and cell viability. UHMCP1 has potential anticancer properties .

HY-148828

LSP-GR3	iGluR	Neurological Disease
LSP-GR3 is a novel chemically-modified RNA oligonucleotides, called splice modulating oligomers (SMOs), which potently and specifically modulate GluR alternative splicing to GluR3-flip expression throughout the CNS.

HY-148828A

LSP-GR3 sodium	iGluR	Neurological Disease
LSP-GR3 sodium is a novel chemically-modified RNA oligonucleotides, called splice modulating oligomers (SMOs), which potently and specifically modulate GluR alternative splicing to GluR3-flip expression throughout the CNS.

HY-174226

RNA binder 2	DNA/RNA Synthesis	Others
RNA binder 2 (Compound 20) is a covalent inhibitor targeting the r(CUG)exp *RNA* in myotonic dystrophy type 1 (DM1). RNA binder 2 binds to RNA via a Hoechst scaffold, blocking MBNL1 binding and restoring normal splicing. RNA binder 2 is promising for research of DM1 .

HY-172552

RECTAS-2.0	DNA/RNA Synthesis	Metabolic Disease
RECTAS-2.0 is a small molecule designed to correct RNA mis-splicing caused by the GLA c.639+919G>A mutation, used for the research of Fabry disease .

HY-112980A

Nusinersen sodium 1 Publications Verification	DNA/RNA Synthesis	Neurological Disease
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 .

HY-112980

Nusinersen 1 Publications Verification	DNA/RNA Synthesis	Neurological Disease
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 .

HY-124648

SMN-C2	DNA/RNA Synthesis	Inflammation/Immunology
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 .

HY-156103

mHTT-IN-2	Huntingtin	Neurological Disease
mHTT-IN-2 (compound 27) is a potent inhibitor (EC₅₀=0.066 μM) of mutant huntingtin (mHTT). mHTT-IN-2 reduces canonical splicing of HTT RNA exons [49-50] and is a splicing regulator of the huntingtin (HTT) gene. mHTT-IN-2 exhibits inhibitory activity in vitro and in vivo in human HD stem cells and mouse BACHD models. mHTT-IN-2 may be used in the study of branaplam-related peripheral neuropathy .

HY-W141374

CB096	DNA/RNA Synthesis	Neurological Disease
CB096 binds the 5′CGG/3′GGC internal loop structure of r(G4C2)exp RNA, interrupts its gain-of-function mechanism, and thus results in the regulation of nucleolar focusing, RNA splicing defects, RNA metabolism, nucleocytoplasmic transport dysfunction, and toxic dipeptide repeats (DPRs) generated by RNA translation. CB096 inhibits the RAN translation with an IC₅₀ of 20 μM in HEK293T cells .

HY-16662

Oncrasin-1	DNA/RNA Synthesis Ras Apoptosis	Cancer
Oncrasin-1 is an RNA polymerase inhibitor. Oncrasin-1 suppresses the phosphorylation of the largest subunit of RNA polymerase II and the expression of intronless reporter genes in sensitive cells. Oncrasin-1 effectively kills various human lung cancer cells with K-Ras mutations. Oncrasin-1 leads to coaggregation of PKCι and splicing factors into megaspliceosomes. Oncrasin-1 induces malfunction in the RNA processing machinery. Oncrasin-1 is an anti-cancer agent and can therefore be studied in research for lung cancer .

HY-122970

1,2-Dihydrotanshinone 1,2-Dihydrotanshinquinone	DNA/RNA Synthesis	Inflammation/Immunology
1,2-Dihydrotanshinone (1,2-Dihydrotanshinquinone) is an abietane diterpene. 1,2-Dihydrotanshinone inhibits the formation of the pathogenic complex formed between (CUG)n-RNA and the splicing-factor muscleblind-like 1 (MBNL1). 1,2-Dihydrotanshinone can be used for the research of myotonic dystrophy type 1 .

HY-155874

ZNL-05-044	CDK	Cancer
ZNL-05-044 is a CDK11 inhibitor with an IC₅₀s of 0.23 μM and 0.27 μM against CDK11A and CDK11B, respectively (NanoBRET assay). ZNL-05-044 leads to G2/M cell cycle arrest and impairs RNA splicing .

HY-129589

Thailanstatin A 2 Publications Verification	ADC Payload	Cancer
Thailanstatin A is an ultra-potent inhibitor of eukaryotic **RNA splicing (IC₅₀**=650 nM). Thailanstatin A exerts effects via non-covalent binding to the SF3b subunit of the U2 snRNA subcomplex of the spliceosome and shows low-nM to sub-nM IC₅₀s against multiple cancer cell lines. Thailanstatin A, a payload for ADCs, is conjugated to the lysines on trastuzumab yielding “linker-less” ADC .

HY-W127770

Pararosaniline hydrochloride Basic red 9	Fluorescent Dye	Infection
Pararosaniline hydrochloride (Basic red 9) is a pH-responsive basic dye, as a biological stain to track certain proteins. The pH of the acidified Pararosaniline hydrochloride reagent has a significant effect on the color and the maximum absorption wavelength (λmax) of the reaction system, with its optimum pH 0.48 and a λmax at 549 nm. Pararosaniline hydrochloride is also a strong modifier of RNA splicing. Pararosaniline hydrochloride has been used in the analysis of SO₂ and formaldehyde and staining of bacteria or other organisms. Pararosaniline hydrochloride is extensively used in industries like textile, printing, paper, cosmetic, and leather .

HY-153918

(R)-SKBG-1	Androgen Receptor	Cancer
(R)-SKBG-1 is a covalent inhibitor targeting the RNA binding protein NONO. (R)-SKBG-1 can reduce the expression of androgen receptor (AR) and its splice variants, inhibiting cancer cell proliferation. (R)-SKBG-1 inhibits androgen receptor expression with IC₅₀ of 3.1 μM and 5.5 μM for AR-FL mRNA and AR-V7 mRNA, respectively. (R)-SKBG-1 interferes with the gene regulatory network of cancer cells and inhibits cancer cell growth by stabilizing the interaction between NONO and mRNA. (R)-SKBG-1 can be used in the study of cancers related to NONO dysfunction, such as prostate cancer .

Cat. No.	Product Name

HY-L917

RNA Binding Library	5,633 compounds
RNA is crucial for the regulation of numerous cellular processes and functions. With the in-depth study of disease mechanisms, processes such as RNA expression, splicing, translation, and stability regulation have become new targets for disease intervention. RNA has provided new therapeutic modalities for metabolic diseases, genetic disorders, and cancer patients, resulting in several innovative drugs. MCE R&D team collected small molecules targeting RNA from the PDB, R-BIND, ROBIN, and internal database as the positive dataset, and non-targeting RNA small molecules from ROBIN as the negative dataset. Based on the GeminiMol pre-trained model, we encoded the molecules and calculated over 1700 molecular descriptors using Mordred as inputs for the model. Subsequently, we employed 13 deep learning models to learn from the data. All of which yielded good training results, with AUROCs greater than 0.75. Ultimately, we selected the Finetune model to screen HY-L901P, which exhibited the best classification performance, achieving an AUROC of 0.82 and a prediction accuracy of 0.76. We then applied filtering based on StaR rules (with at least two of the following properties: cLogP ≥ 1.5, Molar Refractivity ≥ 4, Relative Polar Surface Area ≤ 0.3) to obtain a library containing approximately 5,000 small molecule compounds targeting RNA. This library serves as a valuable tool for screening small molecules that interact with RNA.

RNA Binding Library

5,633 compounds

RNA is crucial for the regulation of numerous cellular processes and functions. With the in-depth study of disease mechanisms, processes such as RNA expression, splicing, translation, and stability regulation have become new targets for disease intervention. RNA has provided new therapeutic modalities for metabolic diseases, genetic disorders, and cancer patients, resulting in several innovative drugs.

MCE R&D team collected small molecules targeting RNA from the PDB, R-BIND, ROBIN, and internal database as the positive dataset, and non-targeting RNA small molecules from ROBIN as the negative dataset. Based on the GeminiMol pre-trained model, we encoded the molecules and calculated over 1700 molecular descriptors using Mordred as inputs for the model. Subsequently, we employed 13 deep learning models to learn from the data. All of which yielded good training results, with AUROCs greater than 0.75. Ultimately, we selected the Finetune model to screen HY-L901P, which exhibited the best classification performance, achieving an AUROC of 0.82 and a prediction accuracy of 0.76. We then applied filtering based on StaR rules (with at least two of the following properties: cLogP ≥ 1.5, Molar Refractivity ≥ 4, Relative Polar Surface Area ≤ 0.3) to obtain a library containing approximately 5,000 small molecule compounds targeting RNA. This library serves as a valuable tool for screening small molecules that interact with RNA.

Cat. No.	Product Name	Target	Research Area