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Results for "

GAA

" in MedChemExpress (MCE) Product Catalog:

27

Inhibitors & Agonists

1

Biochemical Assay Reagents

3

Antibodies

22

Oligonucleotides

Cat. No. Product Name Target Research Areas Chemical Structure
  • HY-150724
    ODN 1018
    2 Publications Verification

    1018 ISS

    Toll-like Receptor (TLR) Inflammation/Immunology
    ODN 1018 (1018 ISS) is a TLR9 agonist and immune modulator. ODN 1018 exhibits adjuvant activity and augments CD8+ T cell responses with LNP-encapsulated OVA peptides. ODN 1018 triggers sustained suppression of allergic airway inflammation. ODN 1018 can be used for the research of allergic asthma and systemic lupus erythematosus .
    ODN 1018
  • HY-132609
    Patisiran sodium
    1 Publications Verification

    Small Interfering RNA (siRNA) Transthyretin (TTR) Neurological Disease
    Patisiran sodium is a double-stranded small interfering RNA that targets a sequence within the transthyretin (TTR) messenger RNA. Patisiran sodium specifically inhibits hepatic synthesis of mutant and wild-type TTR. Patisiran sodium can be used for the research of hereditary TTR amyloidosis .
    Patisiran sodium
  • HY-108753

    AVI 4658

    Arp2/3 Complex Metabolic Disease
    Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide. Eteplirsen can be used for Duchenne muscular dystrophy research .
    Eteplirsen
  • HY-150738
    ODN 2088
    3 Publications Verification

    Toll-like Receptor (TLR) Inflammation/Immunology
    ODN 2088 is a potent TLR3, TLR7 and TLR9 inhibitor. ODN 2088 shows no cytotoxic. ODN 2088 inhibits the release of IFN-α and IL-6 .
    ODN 2088
  • HY-132611

    SRP-4053

    Dystrophin DNA/RNA Synthesis Neurological Disease
    Golodirsen (SRP-4053) is an antisense oligonucleotide of the phophorodiamidate morpholino oligomer (PMO). Golodirsen restores the reading frame of the Duchenne muscular dystrophy (DMD) gene by modifying the splicing process of the pre-mRNA, skipping exon 53. Golodirsen can restore the expression of the anti-myostatin protein. Golodirsen can be used for the research of duchenne muscular dystrophy (DMD) .
    Golodirsen
  • HY-132586

    NS-065/NCNP-01

    Nucleoside Antimetabolite/Analog Dystrophin Metabolic Disease
    Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen has the potential for Duchenne muscular dystrophy (DMD) research .
    Viltolarsen
  • HY-132608

    ISIS-420915 sodium

    Transthyretin (TTR) Neurological Disease
    Inotersen (ISIS-420915) sodium is a 2′-O-methoxyethyl-modified antisense oligonucleotide. Inotersen sodium inhibits the production of transthyretin (TTR) protein by targeting the TTR RNA transcript and reduces the levels of the TTR transcript. Inotersen sodium can be used for the research of hereditary TTR amyloidosis polyneuropathy .
    Inotersen sodium
  • HY-147217

    ISIS 505358

    HBV Infection
    Bepirovirsen is an antisense oligonucleotide targeting all HBV messenger RNAs. Bepirovirsen leads to reductions in HBV-derived RNAs, HBV DNA and viral proteins. Bepirovirsen can be used for the research of chronic HBV infection. Bepirovirsen binding site sequence (GCACTTCGCTTCACCTCTGC) .
    Bepirovirsen
  • HY-150750

    Toll-like Receptor (TLR) Apoptosis Inflammation/Immunology Cancer
    ODN M362, a class C oligodeoxynucleotide, is a TLR-9 agonist and can be used as a vaccine adjuvant. ODN M362 induces cancer cell apoptosis .
    ODN M362
  • HY-150736
    ODN 20844
    1 Publications Verification

    Toll-like Receptor (TLR) Infection Inflammation/Immunology
    ODN 20844, a guanine-modified inhibitory oligonucleotide (INH-ODN), is a TLR7 and TLR9 (Toll-like receptor) inhibitor, and its parent is INH-ODN 2088. ODN 20844 disrupts TLR7- and TLR9-mediated immune cell immune responses. ODN 20844 sequence: 5'-TCCTGGCGc7GGGAAGT-3' .
    ODN 20844
  • HY-148100
    Emapticap pegol
    1 Publications Verification

    NOX-E36

    CCR Inflammation/Immunology Cancer
    Emapticap pegol (NOX-E36) is a inhibitor of pro-inflammatory chemokine C-C motif-ligand 2 (CCL2). Emapticap pegol is a 40-nucleotide oligonucleotide aptamer, displays different Spiegelmers (L-RNA aptamer) isform in human (NOX-E36) and mouse (mNOX-E36). mNOX-E36 is a murine-specific analogue of NOX-E36, an anti-MCP-1 L-RNA aptamer that was previously shown to attenuate liver fibrosis in mice .
    Emapticap pegol
  • HY-145725A

    ISIS 598769; IONIS 598769; BIIB 065; ISIS-DMPK-2.5Rx

    Ser/Thr Kinase Neurological Disease
    Baliforsen (ISIS 5987690) is an antisense oligonucleotide (ASO) that inhibits DMPK mRNA. Baliforsen binds within exon 9 of the human DMPK transcript to promote RNase H1-mediated degradation Baliforsen can be used for the research of myotonic dystrophy type 1 .
    Baliforsen
  • HY-147412

    QR-421a

    Nucleoside Antimetabolite/Analog DNA/RNA Synthesis Neurological Disease
    Ultevursen (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 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 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 can be used for related research on type 2 Usher syndrome and non-syndromic retinitis pigmentosa .
    Ultevursen
  • HY-132596

    SYL1001

    Small Interfering RNA (siRNA) TRP Channel Neurological Disease
    Tivanisiran (SYL1001) is a siRNA used for the study of dry eye disease. Tivanisiran was designed to silence transient receptor potential vanilloid 1 (TRPV1) mRNA .
    Tivanisiran
  • HY-E70183

    EC:3.2.1.20; GAA

    Biochemical Assay Reagents Others
    Lysosomal α-Glucosidase (EC:3.2.1.20) is a γ-amylase with specificity for glycogen and several natural and synthetic oligoglucosides .
    Lysosomal α-Glucosidase
  • HY-132602

    MRG-201

    MicroRNA Inflammation/Immunology
    Remlarsen (MRG-201), a miR-29b mimic, acts a miR-29b agonist. Remlarsen has the potential for preventiong formation of a fibrotic scar or cutaneous fibrosis .
    Remlarsen
  • HY-178202

    Glycosidase Metabolic Disease
    5-C-phenethyl-DNJ is a selective α-glucosidase GAA inhibitor, with its Ki value for rhGAA being 0.81 μM. 5-C-phenethyl-DNJ exhibits extremely high selectivity for GANAB, GBA1, and GBA2. 5-C-phenethyl-DNJ can be used for the study of Pompe disease .
    5-C-phenethyl-DNJ
  • HY-150744

    Toll-like Receptor (TLR) Inflammation/Immunology
    ODN 24888 is an guanine-modified inhibitory oligonucleotides (INH-ODN), shows potent inhibition on TLR7/TLR9-mediated signaling. ODN 24888 impairs IFN-α level and NF-κB activation, inhibits IL-6 release. ODN 24888 involves in immune and inflammatory responses, can be used as a vaccine adjuvant .
    ODN 24888
  • HY-144124

    Glycosidase Others
    5-heptyl-DNJ is a potent GAA agonist with a Ki of 0.0047 μM. 5-C-heptyl-DNJ increases GAA activities by chaperrone effects .
    5-C-heptyl-DNJ
  • HY-RS17242

    Small Interfering RNA (siRNA) Others

    Gaa Mouse Pre-designed siRNA Set A contains three designed siRNAs for Gaa gene (Mouse), as well as a negative control, a positive control, and a FAM-labeled negative control.

    Gaa Mouse Pre-designed siRNA Set A
    Gaa Mouse Pre-designed siRNA Set A
  • HY-RS05196

    Small Interfering RNA (siRNA) Others

    GAA Human Pre-designed siRNA Set A contains three designed siRNAs for GAA gene (Human), as well as a negative control, a positive control, and a FAM-labeled negative control.

    GAA Human Pre-designed siRNA Set A
    GAA Human Pre-designed siRNA Set A
  • HY-RS23696

    Small Interfering RNA (siRNA) Others

    Gaa Rat Pre-designed siRNA Set A contains three designed siRNAs for Gaa gene (Rat), as well as a negative control, a positive control, and a FAM-labeled negative control.

    Gaa Rat Pre-designed siRNA Set A
    Gaa Rat Pre-designed siRNA Set A
  • HY-154995

    Glycosidase Infection
    N-4′-(p-Trifluoromethylphenyl)butyl-DAB (compound 5g) is an agonit of lysosomal acid α-glucosidase (GAA). N-4′-(p-Trifluoromethylphenyl)butyl-DAB increases intracellular GAA activities dose-dependently, in Pompe patient's fibroblasts with the M519V mutation .
    N-4′-(p-Trifluoromethylphenyl)butyl-DAB
  • HY-183723

    Dihydroceramide Desaturase 1 (DES1) Metabolic Disease
    GAA-4OH is a potent and irreversible dihydroceramide desaturase-1 (DES1) inhibitor with an IC50 of 0.6 μM and a Ki of 139.5 nM. GAA-4OH undergoes oxidation to form a reactive iminoquinone that covalently blocks DES1’s catalytic cavity, causing permanent enzyme inactivation. GAA-4OH modulates sphingolipid balance by reducing ceramide-to-dihydroceramide ratios in liver tissue. GAA-4OH improves liver steatosis, inflammation, fibrosis, and reduces pro-inflammatory and pro-fibrogenic gene expression. GAA-4OH can be used for the research of metabolic dysfunction-associated steatotic liver disease (MASLD) .
    GAA-4OH
  • HY-150740

    Toll-like Receptor (TLR) Inflammation/Immunology
    ODN 21595 is a Guanine-Modified TLR7 and TLR9 inhibitor. ODN 21595 inhibits the release of IFN-α and IL-6 with no cytotoxic. ODN 21595 reduces the expression of CD86 and HLA-DR. ODN 21595 has the potential for the research of systemic lupus erythematosus (SLE) .
    ODN 21595
  • HY-182209

    DNA/RNA Synthesis Phosphoramidites Others
    GAA Trimer phosphoramidite is a phosphoramidite that can be used in the synthesis of oligonucleotides.
    GAA Trimer phosphoramidite
  • HY-178200

    Glycosidase Metabolic Disease
    5-C-phenethyl-DNJ hydrochloride is an orally active acid α-glucosidase (GAA) activator. 5-C-phenethyl-DNJ hydrochloride can increase GAA activity in various tissues, particularly in the diaphragm. 5-C-phenethyl-DNJ hydrochloride can be used for the research of metabolic disease, such as Pompe disease .
    5-C-phenethyl-DNJ hydrochloride

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