1. Signaling Pathways
  2. Cytoskeleton
  3. Dystrophin

Dystrophin

Dystrophin is a rod-shaped cytoskeletal protein that connects the intercellular cytoskeleton to the extracellular matrix through an actin-associated glycoprotein complex. This property enables Dystrophin to play a role in sarcolemmal stability during muscle contraction and prevent contraction damage. Dystrophin also mediates cell signaling, such as mechanotransduction and cell adhesion. However, Dystrophin deficiency or mutation (producing internally truncated Dystrophin) will become a predisposing factor for Duchenne muscular dystrophy (DMD). In the muscle tissue of mild and asymptomatic patients, a large number of alternative Dystrophin splicing protein products can be detected. Currently, the methods for restoring Dystrophin mutations rely on virus-mediated restoration or exon skipping. Exon skipping uses antisense oligonucleotides to induce alternative splicing, bypassing the mutated exon to restore the protein reading frame, and converting DMD mutations to Becker muscular dystrophy (BMD) protein mutations.
Dystrophin-related proteins can be divided into three groups (according to subcellular localization): (1) α-dystroglycan located outside the cell; (2) β-dystroglycan, sarcoglycans, and sarcospan located on the plasma membrane; (3) Dystrophin, dystrobrevin, syntrophins, and neuronal nitric oxide synthase located inside the cell. Dystrophin has four major functional domains: the actin-binding amino-terminal domain (ABD1), the central rod domain, the cysteine-rich domain, and the carboxyl terminus. Dystrophin interacts with tubulin and acidic actin filaments through the rod domain, so Dystrophin mutations not only lead to progressive loss of muscle tissue and function, but also cause cardiomyopathy[1][2][3].

Dystrophin Related Products (12):

Cat. No. Product Name Effect Purity Chemical Structure
  • HY-132586A
    Viltolarsen sodium
    Modulator
    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-132584A
    Casimersen sodium
    Inhibitor 99.07%
    Casimersen sodium is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer subclass. Casimersen sodium binds to exon 45 of dystrophin pre-mRNA, restores the open-reading frame (by skipping exon 45) resulting in the production of an internally truncated but functional dystrophin protein. Casimersen sodium can be used for the research of Duchenne muscular dystrophy (DMD).
    Casimersen sodium
  • HY-132611
    Golodirsen
    Modulator
    Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) that specifically targets exon 53 of dystrophin pre-mRNA. Golodirsen can be used for the research of Duchenne muscular dystrophy (DMD).
    Golodirsen
  • HY-145724
    Drisapersen
    Inhibitor
    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.
    Drisapersen
  • HY-101459
    RTC13
    Modulator 99.31%
    RTC13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy (DMD).
    RTC13
  • HY-122631
    TG693
    Modulator 99.95%
    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-150237
    FITC-labeled Drisapersen sodium
    Inhibitor
    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.
    FITC-labeled Drisapersen sodium
  • HY-132584
    Casimersen
    Modulator
    Casimersen (SRP-4045) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer subclass. Casimersen binds to exon 45 of dystrophin pre-mRNA, restores the open-reading frame (by skipping exon 45) resulting in the production of an internally truncated but functional dystrophin protein. Casimersen can be used for the research of Duchenne muscular dystrophy (DMD).
    Casimersen
  • HY-132586
    Viltolarsen
    Modulator
    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-147253A
    Brogidirsen sodium
    Activator
    Brogidirsen sodium exerts the stimulatory function of synthesis of functional dystrophin.
    Brogidirsen sodium
  • HY-163665
    AHR antagonist 8
    Modulator
    AHR antagonist 8 (compound SG-02) is a regulator of utrophin, a homolog of dystrophin, and an AhR antagonist (Kd: 41.68 nM). Studies have shown that 800 nM of AHR antagonist 8 can upregulate utrophin by 2.7 times. AHR antagonist 8 also stimulates increased MyHC expression, suggesting that it has the potential to enhance myogenesis. After ADME evaluation, AHR antagonist 8 also has a certain oral bioavailability.
    AHR antagonist 8
  • HY-123359
    RTC14
    Modulator
    RTC14 is a read-through compound (RTC) that can induce ribosomes to bypass nonsense mutations in mRNA and allow the production of full-length functional proteins. RTC14 has the potential to be used in the research of various genetic disorders, such as nonsense mutations in the ataxia-telangiectasia mutated (ATM) gene and the dystrophin gene.
    RTC14