Search Result
Results for "
silenced
" in MedChemExpress (MCE) Product Catalog:
3
Biochemical Assay Reagents
2
Isotope-Labeled Compounds
Cat. No. |
Product Name |
Target |
Research Areas |
Chemical Structure |
-
- HY-112758
-
|
Liposome
|
Others
|
DLin-KC2-DMA is an ionisable cationic lipid (pKa≈6) that is virtually non-toxic to antigen presenting cells (APCs). DLin-KC2-DMA produces significant siRNA-mediated gene silencing of GAPDH, when binds to lipid nanoparticles (LNP). DLin-KC2-DMA can be used in siRNA delivery studies .
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-
-
- HY-103397
-
|
Antibiotic
DNA Methyltransferase
Parasite
|
Infection
Cancer
|
Nanaomycin A is the first selective DNMT3B inhibitor with an IC50 of 500 nM. Nanaomycin A, a quinone antibiotics, reactivates silenced tumor suppressor genes in human cancer cells . Nanaomycin A inhibits in vitro growth of the human malaria parasite Plasmodium falciparum with an IC80 value of 33.1 nM .
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-
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- HY-154974
-
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Liposome
|
Cancer
|
LNP Lipid-8 (11-A-M) is an ionizable lipid, which can be used for lipid nanoparticles (LNP) to deliver siRNA to T cells without targeting to ligands. LNP LIPs-8 loaded with GFP siRNA (siGFP), and significantly causes GFP gene silencing in mice model .
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- HY-44062
-
|
Histone Methyltransferase
|
Inflammation/Immunology
Cancer
|
G9a-IN-1 (Compound 113) is a G9a protein inhibitor. G9A/EHMT2 is a nuclear histone lysine methyltransferase that catalyzes histone H3 lysine 9 dimethylation (H3K9me2), which is a reversible modification generally associated with transcriptional gene silencing. G9a-IN-1 can be used for the research of autoimmune disorders or cancer .
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-
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- HY-132596A
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SYL1001 sodium
|
TRP Channel
|
Neurological Disease
|
Tivanisiran sodium is a siRNA used for the study of dry eye disease. Tivanisiran sodium was designed to silence transient receptor potential vanilloid 1 (TRPV1) mRNA .
|
-
-
- HY-136869
-
|
Liposome
|
Others
|
MHAPC-Chol, a cationic cholesterol, results in a high gene silencing effect via high cellular association. MHAPC-Chol delivers siRNA to the liver .
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-
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- HY-168088
-
|
HDAC
DNA Methyltransferase
|
Cancer
|
DNMT1/HDAC-IN-1 (compound (R)-23a) is a DNMT1/HDAC dual inhibitor (HDAC1:IC50=0.05 μM), HDAC1 is a major HDAC isoform that interacts with DNMT1 in multiple protein complexes for transcriptional silencing of TSGs. DNMT1/HDAC-IN-1 can reshape the tumor immune microenvironment and induce tumor regression, and effectively reverse cancer-specific epigenetic abnormalities .
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- HY-145523
-
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Liposome
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Others
|
OH-Chol is a cationic cholesterol derivative. OH-Chol can be used for siRNA delivery and gene silencing .
|
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- HY-153479
-
-
-
- HY-153479A
-
-
-
- HY-132596
-
-
-
- HY-135128
-
-
-
- HY-135129
-
|
Histone Methyltransferase
|
Cancer
|
Dot1L-IN-6 is a potent disruptor of telomeric silencing 1-like protein (DOT1L) inhibitor with an IC50 SPA DOT1L of 0.19 nM .
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-
-
- HY-135127
-
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Histone Methyltransferase
|
Cancer
|
Dot1L-IN-4 is a potent disruptor of telomeric silencing 1-like protein (DOT1L) inhibitor with an IC50 SPA DOT1L of 0.11 nM .
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-
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- HY-150409
-
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AUTACs
|
Cancer
|
TSPO ligand-2 is a ligand of AUTAC1. AUTAC1 contains an p-fluorobenzylguanine (FBnG) and a Fumagillol moiety, which can silence endogenous MetAP2 in HeLa cells .
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-
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- HY-P99807
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BCD-100
|
PD-1/PD-L1
|
Cancer
|
Prolgolimab (BCD-100) is a human IgG1 anti-PD-1 monoclonal antibody containing the Fc-silencing 'LALA' mutation. Prolgolimab can be used for the research of advanced melanoma .
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-
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- HY-P10055A
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PSMA-1 TFA
|
PSMA
|
Cancer
|
PSMA targeting peptide TFA (PSMA-1 TFA) is a PSMA targeting peptide (GRFLTGGTGRLLRIS) and can be used for targeted delivery of glucose-regulated protein (GRP)-silencing siRNAs in PCa cells .
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- HY-P99947
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XVR011
|
SARS-CoV
|
Infection
|
Rimteravimab (XVR011) is a bivalent VHH-Fc antibody with potent neutralizing activity with high stability, broad coverage and silenced Fc effector functions against the disease caused by SARS-CoV-2 .
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- HY-125980
-
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p38 MAPK
|
Cancer
|
LX-3 is a selective activator of the p38 MAPK signaling pathway and activates EGFP reporter genes that are silenced by DNA methylation, such as TNF, EGR1, LY6K, and ISG20 .
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- HY-150410
-
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AUTACs
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Cancer
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TSPO ligand-3 is a ligand of AUTAC2. AUTAC2 contains an p-fluorobenzylguanine (FBnG) and an synthetic ligand of FKBP (SLF) moiety, which can cause significant silencing of FKBP12 in HeLa cells .
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-
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- HY-139305
-
|
Liposome
|
Cancer
|
CL4H6 is a pH-sensitive cationic lipid. CL4H6 is the main component of lipid nanoparticles (LNPs), which can be used to target and deliver siRNA, and induces a potent gene-silencing response .
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-
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- HY-161759
-
|
Histone Methyltransferase
|
Metabolic Disease
|
MS152 is an oral bioactive inhibitor of EHMT2/G9a. MS152 reactivats maternally silenced Prader-Willi syndrome (PWS) genes in brain and liver tissues of PWS mouse models .
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-
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- HY-159675
-
|
Liposome
|
Inflammation/Immunology
|
1-A-N is a lipid nanoparticle (LNP) used for in vivo delivery of siRNA. 1-A-N can regulate immune response by delivering siCD45 (siRNA targeting CD45) to T cells and silencing the CD45 gene .
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- HY-101508
-
|
Histone Methyltransferase
|
Cancer
|
GNA002 is a highly potent, specific and covalent EZH2 (Enhancer of zeste homolog 2) inhibitor with an IC50 of 1.1 μM. GNA002 can specifically and covalently bind to Cys668 within the EZH2-SET domain, triggering EZH2 degradation through COOH terminus of Hsp70-interacting protein (CHIP)-mediated ubiquitination. GNA002 efficiently reduces EZH2-mediated H3K27 trimethylation, reactivates polycomb repressor complex 2 (PRC2)-silenced tumor suppressor genes .
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-
- HY-153482
-
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Small Interfering RNA (siRNA)
|
Cancer
|
ATU027 is a siRNA, which silences expression of protein kinase N3 (PKN3) in the vascular endothelium. ATU027 has previously been shown to inhibit local tumor invasion as well as lymph node and pulmonary metastasis in mouse cancer models.
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- HY-153482A
-
|
Small Interfering RNA (siRNA)
|
Cancer
|
ATU027 sodium is a siRNA, which silences expression of protein kinase N3 (PKN3) in the vascular endothelium. ATU027 sodium has previously been shown to inhibit local tumor invasion as well as lymph node and pulmonary metastasis in mouse cancer models.
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- HY-B0106
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UCB L059
|
DNA Methyltransferase
|
Neurological Disease
Cancer
|
Levetiracetam, an antiepileptic agent, binds the synaptic vesicle protein SV2A. Levetiracetam enhances Temozolomide effect on glioblastoma stem cell proliferation and apoptosis. Levetiracetam modulates HDAC levels ultimately silencing MGMT, thus increasing Temozolomide effectiveness. A chemosensitizer agent .
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-
-
- HY-153484A
-
|
VEGFR
Small Interfering RNA (siRNA)
|
Others
|
Bevasiranib sodium is a siRNA designed to silence the genes that produce vascular endothelial growth factor (VEGF). It is widely accepted that vascular endothelial growth factor (VEGF) is a key component in the pathogenesis of choroidal neo-vascularization (CNV), which is a precursor to wet age-related macular degeneration (wet AMD).
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-
-
- HY-153484
-
|
VEGFR
Small Interfering RNA (siRNA)
|
Others
|
Bevasiranib is a siRNA designed to silence the genes that produce vascular endothelial growth factor (VEGF). It is widely accepted that vascular endothelial growth factor (VEGF) is a key component in the pathogenesis of choroidal neo-vascularization (CNV), which is a precursor to wet age-related macular degeneration (wet AMD).
|
-
-
- HY-160852
-
|
Liposome
|
Inflammation/Immunology
|
YSK 12C4 is an ionizable cationic lipid primarily used to enhance siRNA cellular delivery via multifunctional envelope-type nanodevices (MEND). YSK 12C4 promotes siRNA uptake and endosomal escape, effectively silencing genes in human immune cell lines .
|
-
-
- HY-161436
-
|
Small Interfering RNA (siRNA)
|
Others
|
TPA-dT is a thymidine analog with a tris[(2-pyridyl)methyl]amine (TPA) group. TPA-dT can be used to modify siRNAs. The TPA-modified siRNAs remain functionally inactive and is incapable of silencing gene expression until the TPA moiety is removed upon exposure to Cu(I) .
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-
-
- HY-78574
-
|
Others
|
Others
|
N-Benzoylcytidine is a substrate for uracil-cytidine kinase 1 (UCK1) and UCK2. N-Benzoylcytidine can be used to synthesize 2-OH protective groups for solid-phase RNA synthesis, as well as synthetic oligonucleotides for UV induction and targeted gene silencing in zebrafish embryos .
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-
-
- HY-148130A
-
RG6091 sodium; RO7248824 sodium
|
E1/E2/E3 Enzyme
|
Others
|
Rugonersen sodium is a locked-nucleic acid (LNA)- modified antisense oligonucleotides (ASOs), and results in reduction of ubiquitin-protein ligase E3A (UBE3A) silencing. Angelman syndrome (AS) sodium is a severe neurodevelopmental disorder caused by the loss of neuronal E3 ligase UBE3A, Rugonersen has been used for AS reasearch .
|
-
-
- HY-160581
-
|
Liposome
|
Others
|
OH-C-Chol is a cationic liposome that serves as a siRNA delivery vehicle. OH-C-Chol (LP-C) and OH-NC-Chol (LP-NC)/siRNA complexes (lipoplexes) showed better performance than NP-C and NP-NC/siRNA complexes (nanocomplexes), respectively. ) greater gene silencing effect .
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-
-
- HY-152038
-
|
Others
|
Others
|
SPEN-IN-1 (compound X1) is an inhibitor of SPEN which is a protein factor with a Kd value of 47 nM. SPEN-IN-1 has high selectivity for RepA, a 431-nucleotide domain in Xist (a non-coding RNA prototype) that comprises 8.5 units of a GC-rich motif responsible for gene silencing .
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-
-
- HY-B0106S
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UCB L059-d6
|
Isotope-Labeled Compounds
DNA Methyltransferase
|
Neurological Disease
Cancer
|
Levetiracetam-d6 is the deuterium labeled Levetiracetam. Levetiracetam, an antiepileptic agent, binds the synaptic vesicle protein SV2A. Levetiracetam enhances Temozolomide effect on glioblastoma stem cell proliferation and apoptosis. Levetiracetam modulates HDAC levels ultimately silencing MGMT, thus increasing Temozolomide effectiveness. A chemosensitizer agent[1][2].
|
-
-
- HY-B0106S1
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UCB L059-d3
|
DNA Methyltransferase
|
Neurological Disease
Cancer
|
Levetiracetam-d3 is the deuterium labeled Levetiracetam. Levetiracetam, an antiepileptic agent, binds the synaptic vesicle protein SV2A. Levetiracetam enhances Temozolomide effect on glioblastoma stem cell proliferation and apoptosis. Levetiracetam modulates HDAC levels ultimately silencing MGMT, thus increasing Temozolomide effectiveness. A chemosensitizer agent[1][2].
|
-
-
- HY-132588
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ALN-G01
|
Small Interfering RNA (siRNA)
|
Metabolic Disease
|
Lumasiran (ALN-G01), a siRNA product, reduces hepatic oxalate production by targeting glycolate oxidase. By silencing the gene encoding glycolate oxidase, Lumasiran depletes glycolate oxidase and thereby inhibits the synthesis of oxalate, which is the toxic metabolite that is directly associated with the clinical manifestations of Primary hyperoxaluria type 1 (PH1) .
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-
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- HY-147087
-
|
Liposome
|
Cancer
|
YSK 05 is a pH-sensitive cationic lipid. YSK 05 improves the intracellular trafficking of non-viral vectors. YSK 05-MEND shows significantly good gene silencing activity and hemolytic activity. YSK 05 overcomes the suppression of endosomal escape by PEGylation. YSK 05 effectively enhances siRNA delivery both in vitro and in vivo .
|
-
-
- HY-148130
-
RG6091; RO7248824
|
E1/E2/E3 Enzyme
|
Others
|
Rugonersen (RG6091; RO7248824) is a locked-nucleic acid (LNA)- modified antisense oligonucleotides (ASOs), and results in reduction of ubiquitin-protein ligase E3A (UBE3A) silencing. Angelman syndrome (AS) is a severe neurodevelopmental disorder caused by the loss of neuronal E3 ligase UBE3A, Rugonersen has been used for AS reasearch .
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-
-
- HY-162478
-
|
Phosphatase
|
Cancer
|
GR-28 is an inhibitor for small C-terminal domain phosphatase 1 (SCP1). GR-28 inhibits the transcriptional activity of repressor element-1 silencing transcription factor (REST), inhibits the proliferation of glioblastoma cells (IC50 is 2.9 and 10.1 µM, for cells A172 and T98G) .
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-
-
- HY-B0106R
-
|
DNA Methyltransferase
|
Neurological Disease
Cancer
|
Levetiracetam (Standard) is the analytical standard of Levetiracetam. This product is intended for research and analytical applications. Levetiracetam, an antiepileptic agent, binds the synaptic vesicle protein SV2A. Levetiracetam enhances Temozolomide effect on glioblastoma stem cell proliferation and apoptosis. Levetiracetam modulates HDAC levels ultimately silencing MGMT, thus increasing Temozolomide effectiveness. A chemosensitizer agent .
|
-
-
- HY-146724
-
|
Histone Methyltransferase
|
Cancer
|
Dot1L-IN-7 (compound 25) is a potent and selective disruptor of telomeric silencing 1-like protein (DOT1L) inhibitor with an IC50 of 1.0 μM. Dot1L-IN-7 selectively killed Mixed Lineage Leukemia (MLL)-AF9 without showing any effect on the growth of E2A-HLF cells .
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-
-
- HY-147410
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ION-363
|
DNA/RNA Synthesis
Others
|
Neurological Disease
|
Ulefnersen (ION363) is an Antisense Oligonucleotide (ASO) directed against the 6th intron of the fused-in sarcoma (FUS) transcript to silence FUS in a non-allele-specific manner. Ulefnersen can reduce postnatal levels of FUS protein in the brain and spinal cord in disease-relevant mouse model of ALS-FUS , delaying motor neuron degeneration. Ulefnersen can be used in the research of Amyotrophic Lateral Sclerosis (ALS) .
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-
-
- HY-147410A
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ION-363 sodium
|
Others
DNA/RNA Synthesis
|
Neurological Disease
|
Ulefnersen sodium (ION363) is an Antisense Oligonucleotide (ASO) directed against the 6th intron of the fused-in sarcoma (FUS) transcript to silence FUS in a non-allele-specific manner. Ulefnersen sodium can reduce postnatal levels of FUS protein in the brain and spinal cord in disease-relevant mouse model of ALS-FUS , delaying motor neuron degeneration. Ulefnersen sodium can be used in the research of Amyotrophic Lateral Sclerosis (ALS) .
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-
-
- HY-10424A
-
PHA-848125 maleate
|
Ser/Thr Kinase
|
Cancer
|
Milciclib (PHA-848125) maleate is a cyclin-dependent kinase inhibitor that impairs melanoma cell growth and modulates gene expression involved in cell cycle regulation. Milciclib maleate has been shown to significantly affect the expression of various genes, including down-regulating PTTG1, contributing to its antiproliferative activity. Milciclib maleate enhances sensitivity to treatment in p53 mutated melanoma cells when combined with PTTG1 silencing.
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-
-
- HY-43515
-
|
Endogenous Metabolite
|
Metabolic Disease
|
ESI1 is a small molecule epigenetic silencing inhibitor. ESI1 can trigger the formation of nuclear condensates of key lipid metabolism regulators SREBP1/2, concentrating transcriptional co-activators to drive lipid/cholesterol biosynthesis. ESI1 can promote myelin regeneration in demyelinated animal models and facilitate de novo myelination on regenerating CNS axons, reversing age-related declines in cognitive abilities .
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-
-
- HY-137499
-
|
Liposome
Endogenous Metabolite
|
Neurological Disease
|
NT1-O12B, an endogenous chemical and a neurotransmitter-derived lipidoid (NT-lipidoid), is an effective carrier for enhanced brain delivery of several blood-brain barrier (BBB)-impermeable cargos. Doping NT1-O12B into BBB-impermeable lipid nanoparticles (LNPs) gives the LNPs the ability to cross the BBB. NT-lipidoids formulation not only facilitate cargo crossing of the BBB, but also delivery of the cargo into neuronal cells for functional gene silencing or gene recombination .
|
-
Cat. No. |
Product Name |
Type |
-
- HY-112758
-
|
Drug Delivery
|
DLin-KC2-DMA is an ionisable cationic lipid (pKa≈6) that is virtually non-toxic to antigen presenting cells (APCs). DLin-KC2-DMA produces significant siRNA-mediated gene silencing of GAPDH, when binds to lipid nanoparticles (LNP). DLin-KC2-DMA can be used in siRNA delivery studies .
|
-
- HY-147087
-
|
Drug Delivery
|
YSK 05 is a pH-sensitive cationic lipid. YSK 05 improves the intracellular trafficking of non-viral vectors. YSK 05-MEND shows significantly good gene silencing activity and hemolytic activity. YSK 05 overcomes the suppression of endosomal escape by PEGylation. YSK 05 effectively enhances siRNA delivery both in vitro and in vivo .
|
-
- HY-137499
-
|
Drug Delivery
|
NT1-O12B, an endogenous chemical and a neurotransmitter-derived lipidoid (NT-lipidoid), is an effective carrier for enhanced brain delivery of several blood-brain barrier (BBB)-impermeable cargos. Doping NT1-O12B into BBB-impermeable lipid nanoparticles (LNPs) gives the LNPs the ability to cross the BBB. NT-lipidoids formulation not only facilitate cargo crossing of the BBB, but also delivery of the cargo into neuronal cells for functional gene silencing or gene recombination .
|
Cat. No. |
Product Name |
Target |
Research Area |
-
- HY-P10055A
-
PSMA-1 TFA
|
PSMA
|
Cancer
|
PSMA targeting peptide TFA (PSMA-1 TFA) is a PSMA targeting peptide (GRFLTGGTGRLLRIS) and can be used for targeted delivery of glucose-regulated protein (GRP)-silencing siRNAs in PCa cells .
|
-
- HY-P10055
-
PSMA-1
|
Peptides
|
Cancer
|
PSMA-1 is a PSMA targeting peptide (GRFLTGGTGRLLRIS) and can be used for for targeted delivery of glucose-regulated protein (GRP)-silencing siRNAs in PCa cells.?PSMA-1 is selected and polyarginine sequences R6?or R9?were added at the C terminus to generate the CTPs. FITC labeling of the peptide with an aminohexanoic acid (Ahx) linker at the N terminus produced FITC-PSMA-1, to track PSMA binding on PCa cells .?
|
Cat. No. |
Product Name |
Target |
Research Area |
-
- HY-P99807
-
BCD-100
|
PD-1/PD-L1
|
Cancer
|
Prolgolimab (BCD-100) is a human IgG1 anti-PD-1 monoclonal antibody containing the Fc-silencing 'LALA' mutation. Prolgolimab can be used for the research of advanced melanoma .
|
-
- HY-P99947
-
XVR011
|
SARS-CoV
|
Infection
|
Rimteravimab (XVR011) is a bivalent VHH-Fc antibody with potent neutralizing activity with high stability, broad coverage and silenced Fc effector functions against the disease caused by SARS-CoV-2 .
|
Cat. No. |
Product Name |
Category |
Target |
Chemical Structure |
Cat. No. |
Product Name |
Chemical Structure |
-
- HY-B0106S
-
|
Levetiracetam-d6 is the deuterium labeled Levetiracetam. Levetiracetam, an antiepileptic agent, binds the synaptic vesicle protein SV2A. Levetiracetam enhances Temozolomide effect on glioblastoma stem cell proliferation and apoptosis. Levetiracetam modulates HDAC levels ultimately silencing MGMT, thus increasing Temozolomide effectiveness. A chemosensitizer agent[1][2].
|
-
-
- HY-B0106S1
-
|
Levetiracetam-d3 is the deuterium labeled Levetiracetam. Levetiracetam, an antiepileptic agent, binds the synaptic vesicle protein SV2A. Levetiracetam enhances Temozolomide effect on glioblastoma stem cell proliferation and apoptosis. Levetiracetam modulates HDAC levels ultimately silencing MGMT, thus increasing Temozolomide effectiveness. A chemosensitizer agent[1][2].
|
-
Cat. No. |
Product Name |
|
Classification |
-
- HY-112758
-
|
|
Cationic Lipids
|
DLin-KC2-DMA is an ionisable cationic lipid (pKa≈6) that is virtually non-toxic to antigen presenting cells (APCs). DLin-KC2-DMA produces significant siRNA-mediated gene silencing of GAPDH, when binds to lipid nanoparticles (LNP). DLin-KC2-DMA can be used in siRNA delivery studies .
|
-
- HY-154974
-
|
|
Cationic Lipids
|
LNP Lipid-8 (11-A-M) is an ionizable lipid, which can be used for lipid nanoparticles (LNP) to deliver siRNA to T cells without targeting to ligands. LNP LIPs-8 loaded with GFP siRNA (siGFP), and significantly causes GFP gene silencing in mice model .
|
-
- HY-132596A
-
SYL1001 sodium
|
|
siRNAs
|
Tivanisiran sodium is a siRNA used for the study of dry eye disease. Tivanisiran sodium was designed to silence transient receptor potential vanilloid 1 (TRPV1) mRNA .
|
-
- HY-139305
-
|
|
Cationic Lipids
|
CL4H6 is a pH-sensitive cationic lipid. CL4H6 is the main component of lipid nanoparticles (LNPs), which can be used to target and deliver siRNA, and induces a potent gene-silencing response .
|
-
- HY-136869
-
|
|
Cationic Lipids
|
MHAPC-Chol, a cationic cholesterol, results in a high gene silencing effect via high cellular association. MHAPC-Chol delivers siRNA to the liver .
|
-
- HY-145523
-
|
|
Cationic Lipids
|
OH-Chol is a cationic cholesterol derivative. OH-Chol can be used for siRNA delivery and gene silencing .
|
-
- HY-153479
-
|
|
Antisense Oligonucleotides
|
Aganirsen is a 25 mer DNA antisense oligonucleotide, which silences expression of insulin receptor substrate-1 (IRS-1).
|
-
- HY-153479A
-
|
|
Antisense Oligonucleotides
|
Aganirsen sodium is a 25 mer DNA antisense oligonucleotide, which silences expression of insulin receptor substrate-1 (IRS-1).
|
-
- HY-132596
-
SYL1001
|
|
siRNAs
|
Tivanisiran (SYL1001) is a siRNA used for the study of dry eye disease. Tivanisiran was designed to silence transient receptor potential vanilloid 1 (TRPV1) .
|
-
- HY-159675
-
|
|
Cationic Lipids
|
1-A-N is a lipid nanoparticle (LNP) used for in vivo delivery of siRNA. 1-A-N can regulate immune response by delivering siCD45 (siRNA targeting CD45) to T cells and silencing the CD45 gene .
|
-
- HY-153482
-
|
|
siRNAs
|
ATU027 is a siRNA, which silences expression of protein kinase N3 (PKN3) in the vascular endothelium. ATU027 has previously been shown to inhibit local tumor invasion as well as lymph node and pulmonary metastasis in mouse cancer models.
|
-
- HY-153482A
-
|
|
siRNAs
|
ATU027 sodium is a siRNA, which silences expression of protein kinase N3 (PKN3) in the vascular endothelium. ATU027 sodium has previously been shown to inhibit local tumor invasion as well as lymph node and pulmonary metastasis in mouse cancer models.
|
-
- HY-153484A
-
|
|
siRNAs
|
Bevasiranib sodium is a siRNA designed to silence the genes that produce vascular endothelial growth factor (VEGF). It is widely accepted that vascular endothelial growth factor (VEGF) is a key component in the pathogenesis of choroidal neo-vascularization (CNV), which is a precursor to wet age-related macular degeneration (wet AMD).
|
-
- HY-153484
-
|
|
siRNAs
|
Bevasiranib is a siRNA designed to silence the genes that produce vascular endothelial growth factor (VEGF). It is widely accepted that vascular endothelial growth factor (VEGF) is a key component in the pathogenesis of choroidal neo-vascularization (CNV), which is a precursor to wet age-related macular degeneration (wet AMD).
|
-
- HY-148130A
-
RG6091 sodium; RO7248824 sodium
|
|
Antisense Oligonucleotides
|
Rugonersen sodium is a locked-nucleic acid (LNA)- modified antisense oligonucleotides (ASOs), and results in reduction of ubiquitin-protein ligase E3A (UBE3A) silencing. Angelman syndrome (AS) sodium is a severe neurodevelopmental disorder caused by the loss of neuronal E3 ligase UBE3A, Rugonersen has been used for AS reasearch .
|
-
- HY-160581
-
|
|
Cationic Lipids
|
OH-C-Chol is a cationic liposome that serves as a siRNA delivery vehicle. OH-C-Chol (LP-C) and OH-NC-Chol (LP-NC)/siRNA complexes (lipoplexes) showed better performance than NP-C and NP-NC/siRNA complexes (nanocomplexes), respectively. ) greater gene silencing effect .
|
-
- HY-132588
-
ALN-G01
|
|
siRNAs
|
Lumasiran (ALN-G01), a siRNA product, reduces hepatic oxalate production by targeting glycolate oxidase. By silencing the gene encoding glycolate oxidase, Lumasiran depletes glycolate oxidase and thereby inhibits the synthesis of oxalate, which is the toxic metabolite that is directly associated with the clinical manifestations of Primary hyperoxaluria type 1 (PH1) .
|
-
- HY-147087
-
|
|
Cationic Lipids
|
YSK 05 is a pH-sensitive cationic lipid. YSK 05 improves the intracellular trafficking of non-viral vectors. YSK 05-MEND shows significantly good gene silencing activity and hemolytic activity. YSK 05 overcomes the suppression of endosomal escape by PEGylation. YSK 05 effectively enhances siRNA delivery both in vitro and in vivo .
|
-
- HY-148130
-
RG6091; RO7248824
|
|
Antisense Oligonucleotides
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Rugonersen (RG6091; RO7248824) is a locked-nucleic acid (LNA)- modified antisense oligonucleotides (ASOs), and results in reduction of ubiquitin-protein ligase E3A (UBE3A) silencing. Angelman syndrome (AS) is a severe neurodevelopmental disorder caused by the loss of neuronal E3 ligase UBE3A, Rugonersen has been used for AS reasearch .
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- HY-147410
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ION-363
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Antisense Oligonucleotides
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Ulefnersen (ION363) is an Antisense Oligonucleotide (ASO) directed against the 6th intron of the fused-in sarcoma (FUS) transcript to silence FUS in a non-allele-specific manner. Ulefnersen can reduce postnatal levels of FUS protein in the brain and spinal cord in disease-relevant mouse model of ALS-FUS , delaying motor neuron degeneration. Ulefnersen can be used in the research of Amyotrophic Lateral Sclerosis (ALS) .
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- HY-147410A
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ION-363 sodium
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Antisense Oligonucleotides
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Ulefnersen sodium (ION363) is an Antisense Oligonucleotide (ASO) directed against the 6th intron of the fused-in sarcoma (FUS) transcript to silence FUS in a non-allele-specific manner. Ulefnersen sodium can reduce postnatal levels of FUS protein in the brain and spinal cord in disease-relevant mouse model of ALS-FUS , delaying motor neuron degeneration. Ulefnersen sodium can be used in the research of Amyotrophic Lateral Sclerosis (ALS) .
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- HY-137499
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Cationic Lipids
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NT1-O12B, an endogenous chemical and a neurotransmitter-derived lipidoid (NT-lipidoid), is an effective carrier for enhanced brain delivery of several blood-brain barrier (BBB)-impermeable cargos. Doping NT1-O12B into BBB-impermeable lipid nanoparticles (LNPs) gives the LNPs the ability to cross the BBB. NT-lipidoids formulation not only facilitate cargo crossing of the BBB, but also delivery of the cargo into neuronal cells for functional gene silencing or gene recombination .
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