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dystrophy

" in MedChemExpress (MCE) Product Catalog:

91

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4

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5

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29

Oligonucleotides

Targets Recommended:
Cat. No. Product Name Target Research Areas Chemical Structure
  • HY-14415
    SR8278
    Maximum Cited Publications
    15 Publications Verification

    REV-ERB Neurological Disease Metabolic Disease
    SR8278 is a REV-ERBα antagonist and inhibits the REV-ERBα transcriptional repression activity with an EC50 of 0.47 μM. SR8278 is used to regulate the metabolism in organisms and study biological rhythm. SR8278 also can be used for the research of Duchenne muscular dystrophy and Alzheimer's disease .
    SR8278
  • HY-14842
    Givinostat
    10+ Cited Publications

    ITF-2357

    HDAC Inflammation/Immunology Cancer
    Givinostat (ITF-2357) is a HDAC inhibitor with an IC50 of 198 and 157 nM for HDAC1 and HDAC3, respectively. Givinostat can be used for Duchenne muscular dystrophy (DMD) research. Givinostat can penetrate the blood-brain barrier (BBB) .
    Givinostat
  • HY-W015828
    Ompenaclid
    5+ Cited Publications

    RGX-202; 3-Guanidinopropionic acid

    Apoptosis Endogenous Metabolite Cancer
    Ompenaclid (RGX-202) is an oral small-molecule SLC6A8 transporter inhibitor. Ompenaclid robustly inhibits creatine import in vitro and in vivo, reduces intracellular phosphocreatine and ATP levels, and induces tumor apoptosis. Ompenaclid can be used for the research of cancer and duchenne muscular dystrophy .
    Ompenaclid
  • HY-109017

    VBP15

    Glucocorticoid Receptor Mineralocorticoid Receptor NF-κB Inflammation/Immunology
    Vamorolone (VBP15) is a first-in-class, orally active dissociative steroidal anti-inflammatory agent and membrane-stabilizer. Vamorolone improves muscular dystrophy without side effects. Vamorolone shows potent NF-κB inhibition and substantially reduces hormonal effects .
    Vamorolone
  • 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-148799

    EDG-5506

    Myosin Others
    Sevasemten is an orally active, selective allosteric inhibitor of skeletal muscle myosin that protects skeletal muscle from contraction-induced injury. Sevasemten decreases muscle damage biomarkers and fibrosis while increasing muscle strength and activity in in Duchenne muscular dystrophy disease models .
    Sevasemten
  • HY-W017540
    Cyclocreatine
    1 Publications Verification

    ATP Synthase Cardiovascular Disease Neurological Disease Metabolic Disease Cancer
    Cyclocreatine, a creatine analogue, acts as a brain-penetrant and potent bioenergetic protective agent by providing high levels of ATP. Cyclocreatine can be phosphorylated and dephosphorylated by creatine kinases. Cyclocreatine suppresses creatine metabolism ameliorating the cognitive, autistic and epileptic phenotype in a mouse model of creatine transporter defciency. Cyclocreatine protects against ischemic injury and enhances cardiac recovery during early reperfusion in dogs and rats. Cyclocreatine decreases plaque-adjacent neuronal dystrophy in TREM2-deficient mice with amyloid-β pathology. Cyclocreatine is proming for research of ischemic heart disease, cardiovascular diseases, Alzheimer’s disease and other neurodegenerative diseases associated with microglial dysfunction, prostate cancer .
    Cyclocreatine
  • 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-110155
    LM11A-31 dihydrochloride
    5+ Cited Publications

    Neurotensin Receptor Neurological Disease
    LM11A-31 dihydrochloride, a non-peptide p75 NTR (neurotrophin receptor p75) modulator, is an orally active and potent proNGF (nerve growth factor) antagonist. LM11A-31 dihydrochloride is an amino acid derivative with high blood-brain barrier permeability and blocks p75-mediated cell death. LM11A-31 dihydrochloride reverses cholinergic neurite dystrophy in Alzheimer's disease mouse models with mid- to late-stage disease progression .
    LM11A-31 dihydrochloride
  • HY-P990051

    AOC-1001 Antibody; AOC-1044 Antibody

    Transferrin Receptor Neurological Disease Inflammation/Immunology
    Delpacibart is a humanized IgG1κ monoclonal antibody targeting the transferrin receptor TFRC. Delpacibart can be conjugated with the phosphorodiamidate morpholino oligonucleotide (PMO) Zotadirsen (HY-177972), which targets exon 44 of the dystrophin gene, to synthesize the antibody-oligonucleotide conjugate (AOC) Delpacibart zotadirsen (HY-177564). Delpacibart is suitable for use in Duchenne muscular dystrophy (DMD44) research .
    Delpacibart
  • 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-119850

    ARM036; S44121

    Calcium Channel Cardiovascular Disease
    Aladorian (ARM036; S44121) is a non-peptidic ryanodine receptor 2 (RyR2) stabilizer. Aladorian stabilizes RyR2 channels and rectifies abnormal Ca²⁺ handling in cardiomyocytes. Aladorian improves cardiomyocyte Ca²⁺ homeostasis independent of dystrophin restoration. Aladorian attenuates early cardiomyopathy and enhances left ventricular function in a canine muscular dystrophy model. Aladorian can be used for the research of heart failure, Duchenne muscular dystrophy-associated cardiomyopathy and muscular dystrophy .
    Aladorian
  • HY-117088
    LM11A-31
    5+ Cited Publications

    Neurokinin Receptor Neurological Disease
    LM11A-31, a non-peptide p75 NTR (neurotrophin receptor p75) modulator, is an orally active and potent proNGF (nerve growth factor) antagonist. LM11A-31 is an amino acid derivative with high blood-brain barrier permeability and blocks p75-mediated cell death. LM11A-31 reverses cholinergic neurite dystrophy in Alzheimer's disease mouse models with mid- to late-stage disease progression .
    LM11A-31
  • HY-127137

    Adenylosuccinate; Aspartyl adenylate

    Endogenous Metabolite Metabolic Disease
    Adenylosuccinic acid (Adenylosuccinate; Aspartyl adenylate) is a purine ribonucleoside monophosphate and plays a role in nucleotide cycle metabolite. Adenylosuccinic acid can be converted into fumaric acid through adenylosuccinate lyase. Adenylosuccinic acid has the potential for the study of duchenne muscular dystrophy(DMD) .
    Adenylosuccinic acid
  • HY-111424
    BDP9066
    2 Publications Verification

    Cdc42-binding kinase Cancer
    BDP9066 is a potent and selective myotonic dystrophy-related Cdc42-binding kinase MRCK inhibitor with an IC50 of 64 nM for MRCKβ in SCC12 cells, Ki values of 0.0136 nM and 0.0233 nM for MRCKα/β in house determinations, respectively. BDP9066 has therapeutic effect on skin cancer by reducing substrate phosphorylation.
    BDP9066
  • HY-P99370
    Landogrozumab
    1 Publications Verification

    LY2495655; Anti-GDF8 / Myostatin Reference Antibody (landogrozumab)

    TGF-beta/Smad Inflammation/Immunology Cancer
    Landogrozumab (LY2495655) is an humanized anti-myostatin monoclonal antibody. Landogrozumab effectively improves muscle volume, hand grip strength and function. Landogrozumab can be used for the research of muscle wasting disease .
    Landogrozumab
  • HY-147332

    Liposome Neurological Disease
    TCL053 is an ionizable lipid carrier and used to introduce active components, in particular nucleic acids, into cells with excellent efriciency. TCL053, together with DPPC (Dipalmitoylphosphatidylcholine), PEG-DMG (Polyethylene glycoldimyristoyl glycerol), and cholesterol, forms lipid nanoparticle (LNP) which is able to deliver Cas9 mRNA and sgRNA into skeletal muscle .
    TCL053
  • HY-P99857

    PF-06252616

    TGF-beta/Smad Metabolic Disease
    Domagrozumab is an anti-myostatin humanized monoclonal antibody with a KD value of 2.6 pM for human myostatin. Domagrozumab induces muscle anabolic activity. Domagrozumab can be used in research of duchenne muscular dystrophy (DMD) .
    Domagrozumab
  • HY-17614

    SMT C1100; BMN 195; VOX-C1100

    Cytochrome P450 Others
    Ezutromid (SMT C1100) is a first-in-class, orally active benzoxazole utrophin modulator with an EC50 of 0.91 μM. Ezutromid can be used for the research Duchenne muscular dystrophy (DMD). Ezutromid inhibits CYP1A2 enzymic activity in human liver microsomes (HLM) with an IC50 of 5.4 μM .
    Ezutromid
  • HY-P990993

    GYM-329, RG6237, RG-70240

    TGF-beta/Smad Inflammation/Immunology
    Emugrobart (GYM-329, RG6237, RG-70240) is a humanized IgG1κ antibody targeting myostatin (Myostatin; GDF8). Emugrobart binds to pro-myostatin and latent myostatin, blocking their cleavage into mature myostatin; it also has a clearance function, which transports bound myostatin for degradation and allows for cyclic reuse. Emugrobart enhances muscle strength in mouse models of muscle atrophy and increases muscle mass in cynomolgus monkeys. Emugrobart can be used in research on spinal muscular atrophy and facioscapulohumeral muscular dystrophy .
    Emugrobart
  • HY-145724

    Kyndrisa; GSK2402968A; PRO051

    DNA/RNA Synthesis Dystrophin Neurological Disease
    Drisapersen (Kyndrisa) is a 2 '-O-methyl phosphorothioate RNA antisense oligonucleotide that induces exon 51 skipping. Drisapersen induces skipping of exon 51 during Dystrophin pre-mRNA splicing, allowing the synthesis of partially functional Dystrophin. Drisapersen can be used in research related to Duchenne muscular dystrophy .
    Drisapersen
  • 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-148457

    Izervay

    Complement System Infection Neurological Disease Inflammation/Immunology
    Avacincaptad pegol (Izervay) is a selective inhibitor targeting complement component C5, and is a pegylated ribonucleic acid aptamer. Avacincaptad pegol inhibits the cleavage of C5 into pro-inflammatory C5a and C5b, which forms the membrane attack complex (C5b-9), thereby reducing inflammatory cell recruitment and retinal cell damage. Avacincaptad pegol can slow the growth of geographic atrophy (GA) lesions and reduce the risk of persistent vision loss. Avacincaptad pegol can be used in research of geographic atrophy associated with age-related macular degeneration (AMD) and has been approved by the FDA .
    Avacincaptad pegol
  • HY-125222

    Dynamin Others
    Drp1-IN-1 is a dynamin-1-like protein (Drp1) inhibitor with an IC50 of 0.91 μM. Drp1 mediates the fission of the outer mitochondrial membrane. Drp1-IN-1 can be used to study diseases associated with mitochondrial dysfunction .
    Drp1-IN-1
  • HY-P99588
    Stamulumab
    1 Publications Verification

    MYO-029

    TGF-β Receptor Metabolic Disease
    Stamulumab (MYO-029) is a recombinant human IgG1λ antibody that binds to myostatin and neutralizes its activity by preventing binding to its endogenous high-affinity receptor ActRIIB. Stamulumab leads to muscle fiber hypertrophy and not hyperplasia in SCID mice. Stamulumab has the potential for Becker muscular dystrophy (BMD), facioscapulohumeral dystrophy (FSHD), and limb-girdle muscular dystrophy (LGMD) research .
    Stamulumab
  • HY-132611A

    SRP-4053 sodium

    DNA/RNA Synthesis Dystrophin Neurological Disease
    Golodirsen (SRP-4053) sodium is an antisense oligonucleotide of the phophorodiamidate morpholino oligomer (PMO). Golodirsen sodium restores the reading frame of the Duchenne muscular dystrophy (DMD) gene by modifying the splicing process of the pre-mRNA, skipping exon 53. Golodirsen sodium can restore the expression of the anti-myostatin protein. Golodirsen sodium can be used for the research of duchenne muscular dystrophy (DMD) .
    Golodirsen sodium
  • HY-152219

    CDK Infection Cancer
    CLK1-IN-2 is metabolically stable Clk1 inhibitor. CLK1-IN-2 has selectivity for Clk1 with an IC50 value of 1.7 nM. CLK1-IN-2 can be used for the research of tumour, Duchenne's muscular dystrophy and viral infections such as HIV-1 and influenza .
    CLK1-IN-2
  • HY-134377

    ASP0367; MA-0211

    PPAR Metabolic Disease
    Bocidelpar (ASP0367; MA-0211) is a selective, orally active PPARδ modulator. Bocidelpar activates the PPARδ downstream signaling pathway, upregulates the expression of target genes such as ABCA1 and ACAA2. Bocidelpar then promotes fatty acid oxidation (FAO) and mitochondrial biogenesis, and improves mitochondrial dysfunction. Bocidelpar can improve mitochondrial biogenesis and function in muscle cells. Bocidelpar is mainly used in the study of mitochondrial dysfunction diseases such as primary mitochondrial myopathy (PMM) and Duchenne muscular dystrophy (DMD) .
    Bocidelpar
  • HY-16361A

    CGP3466B; CGP3446 maleate; TCH346 maleate

    Apoptosis Neurological Disease Metabolic Disease Inflammation/Immunology
    Omigapil maleate (CGP3466B), an orally active GAPDH nitrosylation inhibitor, abrogates Aβ1-42-induced tau acetylation, memory impairment, and locomotor dysfunction in mice. Omigapil maleate has the potential for the research of Alzheimer's disease. Omigapil maleate is a apoptosis inhibitor. Omigapil maleate can be used for the research of congenital muscular dystrophy (CMD). Omigapil maleate is a click chemistry reagent, it contains an Alkyne group and can undergo copper-catalyzed azide-alkyne cycloaddition (CuAAc) with molecules containing Azide groups .
    Omigapil maleate
  • HY-18102
    GLPG0492
    5+ Cited Publications

    Androgen Receptor Neurological Disease
    GLPG0492 is an orally active, non-steroidal selective androgen receptor modulator. GLPG0492 exerts functional transactivation by binding to the ligand-binding domain of the receptor, exhibiting preferential partial agonist activity in muscle and bone tissues with low activity in reproductive tissues. GLPG0492 effectively counteracts muscle atrophy-related pathways, significantly enhances muscle strength, maintains motor ability, reduces fibrosis and improves electrophysiological parameters. GLPG0492 prevents immobilization-induced muscle atrophy and regulates muscle mass homeostasis, serving as a valuable tool compound for studies on Duchenne muscular dystrophy, muscle loss and various types of disuse musculoskeletal atrophy .
    GLPG0492
  • HY-P10242

    TGF-beta/Smad Others
    Myostatin inhibitory peptide 7 is a 23 amino acids peptide, which is derived from amino acids 21 to 43 of the mouse myostatin prodomain. Myostatin inhibitory peptide 7 inhibits myostatin with a Kd of 29.7 nM. Myostatin inhibitory peptide 7 can be used for researches of muscle atrophic disorders .
    Myostatin inhibitory peptide 7
  • HY-132586A

    NS-065/NCNP-01 sodium

    Nucleoside Antimetabolite/Analog Dystrophin Metabolic Disease
    Viltolarsen (NS-065/NCNP-01) sodium is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen sodium 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 sodium has the potential for Duchenne muscular dystrophy (DMD) research .
    Viltolarsen sodium
  • HY-122631

    CDK Dystrophin Others
    TG693 is an orally active inhibitor of CLK1. TG693 regulates the mutated exon 31 of the dystrophin gene in vivo. TG693 is used in Duchenne muscular dystrophy (DMD) research .
    TG693
  • HY-147253

    NS 089; NCNP 02

    DNA/RNA Synthesis Neurological Disease
    Brogidirsen (NS 089; NCNP 02) is a a dual-targeting antisense oligonucleotide. Brogidirsen can induce dystrophin protein experession. Brogidirsen can be used for the research of Duchenne muscular dystrophy .
    Brogidirsen
  • HY-177564

    Del-zota; AOC 1044

    Antibody-Oligonucleotide Conjugates (AOCs) Transferrin Receptor Dystrophin Neurological Disease
    Delpacibart zotadirsen (Del-zota), an antibody oligonucleotide conjugate (AOC), consists of a monoclonal antibody (Delpacibart) (HY-P990051) that binds to the transferrin receptor 1 (TfR1) conjugated to a phosphorodiamidate morpholino conjugate (PMO), Delpacibart zotadirsen is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene and enable production of near-full length dystrophin. Delpacibart zotadirsen is used for the study of myotonic dystrophy type 1 (DM1) .
    Delpacibart zotadirsen
  • HY-110155A

    Drug Isomer Neurological Disease
    (Rac)-LM11A-31 dihydrochloride is an isomer of LM11A-31 dihydrochloride. LM11A-31 dihydrochloride, a non-peptide p75 NTR (neurotrophin receptor p75) modulator, is an orally active and potent proNGF (nerve growth factor) antagonist .
    (Rac)-LM11A-31 dihydrochloride
  • HY-101459

    Dystrophin Metabolic Disease
    RTC13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy (DMD) .
    RTC13
  • HY-122970

    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 .
    1,2-Dihydrotanshinone
  • HY-127137A

    Adenylosuccinate tetraammonium; Aspartyl adenylate tetraammonium

    Endogenous Metabolite Metabolic Disease
    Adenylosuccinic acid tetraammonium (Adenylosuccinate; Aspartyl adenylate) is an orally active purine ribonucleoside monophosphate and plays a role in nucleotide cycle metabolite. Adenylosuccinic acid tetraammonium can be converted into fumaric acid through adenylosuccinate lyase. Adenylosuccinic acid tetraammonium has the potential for the study of duchenne muscular dystrophy(DMD) .
    Adenylosuccinic acid tetraammonium
  • HY-161050

    HDAC Apoptosis Cancer
    YSR734 (Compound 21) is a covalent HDAC inhibitor with IC50 values of 110 nM, 154 nM, and 143 nM for HDAC1, HDAC2, and HDAC3, respectively. YSR734 can induce apoptosis in leukemia cells. YSR734 can induce myoblast differentiation and is used in the study of Duchenne muscular dystrophy .
    YSR734
  • HY-16918A

    SBi4211 dimethanesulfonate

    Calmodulin Inflammation/Immunology Cancer
    Heptamidine dimethanesulfonate (SBi4211 dimethanesulfonate) is a potent Pentamidine-related inhibitor of the calcium-binding protein S100B (Kd=6.9 μM), selectively kills melanoma cells with S100B over those without S100B . Heptamidine is a useful tool for the investigation of Myotonic dystrophy (DM) .
    Heptamidine dimethanesulfonate
  • HY-177454

    ADC Linker Others
    Basivarsen linker is a linker used in HY-177452 Zeleciment basivarsen for coupling a TfR1-binding Fab (HY-P990780 Zeleciment) and an antisense oligonucleotide. Zeleciment basivarsen is an antibody-oligonucleotide conjugate (AOC) designed to target mutant nuclear myotonic dystrophy protein kinase (DMPK) RNA for RHase H-mediated degradation to correct splicing. It is used for the study of myotonic dystrophy type 1 (DM1).
    Basivarsen linker
  • HY-111423

    Cdc42-binding kinase Cancer
    BDP8900 is a potent and selective inhibitor of myotonic dystrophy-related Cdc42-binding kinases (MRCKα and MRCKβ). BDP8900 reduces substrate phosphorylation, leading to morphological changes, motility inhibition and invasiveness of cancer cells .
    BDP8900
  • HY-114739

    PPAR Metabolic Disease
    MA-0204 is a potent, highly selective and orally available peroxisome proliferator activated receptor δ (PPARδ) modulator with EC50s of 0.4 nM, 7.9 nM and 10 nM for human, mouse and rat PPARδ, respectively. Potential treatment for Duchene Muscular Dystrophy (DMD) .
    MA-0204
  • HY-109134

    TAS-205 free base

    Prostaglandin Receptor Metabolic Disease Inflammation/Immunology
    Pizuglanstat (Compound 3; TAS-205 free base) is an orally active prostaglandin D synthase inhibitor with an IC50 of 76 nM for human hematopoietic prostaglandin D synthase. Pizuglanstat inhibits the synthesis of PGD2. Pizuglanstat improves experimental allergic rhinitis. Pizuglanstat can be used in the study of muscle regenerative diseases such as muscular dystrophy .
    Pizuglanstat
  • HY-147774

    Cathepsin Others
    Cathepsin K inhibitor 6 (compound 19) is an inhibitor of cathepsin K (Cat K) with an IC50 of 17 nM. Cathepsin K inhibitor 6 also has inhibitory effects on other isoforms, with IC50s of 0.05 μM (Cat L) and 0.3 μM (Cat B), respectively .
    Cathepsin K inhibitor 6
  • HY-111424A

    Cdc42-binding kinase Cancer
    (R)-BDP9066 is an isomer of BDP9066 and has lower activity compared to BDP9066. BDP9066 is a potent inhibitor of myotonic dystrophy kinase-related Cdc42-binding kinase (MRCK). (R)-BDP9066 is derived from patent WO2019034890A1 (E118) .
    (R)-BDP9066
  • HY-153405

    Biochemical Assay Reagents Phosphoramidites Others
    Activated DPG Subunit can be used in the synthesis of exon jumping oligomer conjugates. The oligomer conjugates complement selected target sites in the human anti-muscular atrophy protein gene and induce exon 51 jumping. It can be used for research of muscular dystrophy .
    Activated DPG Subunit
  • HY-14842R

    ITF-2357 (Standard)

    HDAC Reference Standards Inflammation/Immunology Cancer
    Givinostat (Standard) is the analytical standard of Givinostat. This product is intended for research and analytical applications. Givinostat (ITF-2357) is a HDAC inhibitor with an IC50 of 198 and 157 nM for HDAC1 and HDAC3, respectively. Givinostat can be used for Duchenne muscular dystrophy (DMD) research .
    Givinostat (Standard)
  • HY-153406

    Biochemical Assay Reagents Others
    Activated T Subunit can be used in the synthesis of exon jumping oligomer conjugates. The oligomer conjugates complement selected target sites in the human anti-muscular atrophy protein gene and induce exon 51 jumping. Activated T subunit can be used for research of muscular dystrophy .
    Activated T Subunit

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