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muscular

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30

Oligonucleotides

Cat. No. Product Name Classification
  • HY-112980
    Nusinersen
    4 Publications Verification

    Antisense Oligonucleotides
    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-108753

    AVI 4658

    Antisense Oligonucleotides
    Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide. Eteplirsen can be used for Duchenne muscular dystrophy research .
  • HY-112980A
    Nusinersen sodium
    4 Publications Verification

    Antisense Oligonucleotides
    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-132611

    SRP-4053

    Antisense Oligonucleotides
    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) .
  • HY-132586

    NS-065/NCNP-01

    Antisense Oligonucleotides
    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 .
  • HY-147332

    Cationic Lipids
    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 .
  • HY-145724

    Kyndrisa; GSK2402968A; PRO051

    Antisense Oligonucleotides
    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 .
  • HY-108753A

    AVI 4658 sodium

    Antisense Oligonucleotides
    Eteplirsen (AVI 4658) sodium is a synthetic antisense oligonucleotide that induces dystrophin production. Eteplirsen (AVI 4658) sodium promotes exon 51 skipping in Duchenne muscular dystrophy patients and can be used in Duchenne muscular dystrophy research .
  • HY-132611A

    SRP-4053 sodium

    Antisense Oligonucleotides
    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) .
  • HY-132586A

    NS-065/NCNP-01 sodium

    Antisense Oligonucleotides
    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 .
  • HY-145969

    3'-O-Me-m7G(5')ppp(5')G

    Cap Analogs
    β-S-ARCA (3'-O-Me-m7G(5')ppp(5')G) is a mRNA 7-methylguanosine (m7G) cap analog carrying a phosphorothioate (PS) moiety. β-S-ARCA binds eIF4E via electrostatic interactions between its β-sulfur atom and positively charged Arg and Lys residues in the protein binding site. β-S-ARCA prevents the decapping by Dcp2, increases the mRNA half-life, enhances cap-dependent translation, and increases protein expression in cells. β-S-ARCA has been applied in researching experimental mRNA-based anticancer vaccines .
  • HY-147253

    NS 089; NCNP 02

    Antisense Oligonucleotides
    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 .
  • HY-127137A

    Adenylosuccinate tetraammonium; Aspartyl adenylate tetraammonium

    Nucleotide Analogs
    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) .
  • HY-145724A

    Kyndrisa sodium; GSK2402968A sodium; PRO051 sodium

    Antisense Oligonucleotides
    Drisapersen sodium, a antisense oligonucleotide, induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthesis of partially functional dystrophin in Duchenne muscular dystrophy (DMD) patients with amenable mutations.
  • HY-132585A

    SRP-5051 sodium

    Antisense Oligonucleotides
    Vesleteplirsen (SRP-5051) sodium is a next-generation antisense oligonucleotide of peptide phosphorodiamidate morpholino oligomer (PPMO). Vesleteplirsen targets exon 51 skipping in Duchenne muscular dystrophy (DMD) .
  • HY-153405

    Phosphoramidites Other Phosphoramidite
    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 .
  • HY-177661

    BIIB115; ION306

    Antisense Oligonucleotides
    Salanersen is an antisense oligonucleotide targeted to survival motor neuron 2 (SMN2). It is used for the study of spinal muscular atrophy (SMA).
  • HY-177659

    Antisense Oligonucleotides
    Rimigorsen is an antisense oligonucleotide that induces skipping of exon 44 of the pre-mRNA encoding dystrophin in a Duchenne muscular dystrophy (DMD)
  • HY-176754

    Cationic Lipids
    TCL065 is an ionizable lipid with a pKa of 6.3. TCL065 can be used to generate lipid nanoparticles (LNPs) for the delivery of mRNA as well as single-guide RNA (sgRNA) both in vitro and in vivo. TCL065-containing LNPs can studied in research for Duchenne muscular dystrophy and gene-editing technologies .
  • HY-177971

    siRNAs
    Braxlosiran, one of the components of the AOC Delpacibart braxlosiran (HY-177566), is composed of a siRNA targeting DUX4 (dual homologous box protein) and SMCC linker (HY-42360). Abnormal expression of the DUX4 protein leads to changes in gene expression in muscle cells, and these changes are associated with the loss of muscle function in facioscapulohumeral muscular dystrophy (FSHD) .
  • HY-132592A

    WVE-210201 sodium

    Antisense Oligonucleotides
    Suvodirsen sodium induces exon 51 skipping and has the potential for study Duchenne muscular dystrophy (DMD) .
  • HY-132592

    WVE-210201

    Antisense Oligonucleotides
    Suvodirsen (WVE-210201) is a oligonucleotide. Suvodirsen has the potential for study Duchenne muscular dystrophy (DMD) .
  • HY-153404

    Phosphoramidites Other Phosphoramidite
    Activated C 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 .
  • HY-153324

    Antisense Oligonucleotides
    PS220 sodium is an antisense RNA oligonucleotides. PS220 sodium can be used for research ofstudying muscular dystrophy .
  • HY-177659A

    Antisense Oligonucleotides
    Rimigorsen sodium is an antisense oligonucleotide that induces skipping of exon 44 of the pre-mRNA encoding dystrophin in a Duchenne muscular dystrophy (DMD)
  • HY-147253A

    NS 089 sodium; NCNP 02 sodium

    Antisense Oligonucleotides
    Brogidirsen (NS 089; NCNP 02) sodium is a a dual-targeting antisense oligonucleotide. Brogidirsen sodium can induce dystrophin protein experession. Brogidirsen sodium can be used for the research of Duchenne muscular dystrophy .
  • HY-150237

    Antisense Oligonucleotides
    FITC-labeled Drisapersen (sodium) is Drisapersen labeled with FITC. Drisapersen, a antisense oligonucleotide, induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthesis of partially functional dystrophin in Duchenne muscular dystrophy (DMD) patients with amenable mutations.
  • HY-185321

    Antisense Oligonucleotides
    Tacadirsen is an oligonucleotide that can promote the synthesis of functional dystrophin synthesis, and is used for the research of Duchenne muscular dystrophy.
  • HY-185321A

    Antisense Oligonucleotides
    Tacadirsen sodium is an oligonucleotide that can promote the synthesis of functional dystrophin synthesis, and is used for the research of Duchenne muscular dystrophy.
  • HY-177661A

    BIIB115 sodium; ION306 sodium

    Antisense Oligonucleotides
    Salanersen sodium is an antisense oligonucleotide targeted to survival motor neuron 2 (SMN2). It is used for the study of spinal muscular atrophy (SMA).

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