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Results for "

myotonic dystrophy type 1

" in MedChemExpress (MCE) Product Catalog:

7

Inhibitors & Agonists

1

Natural
Products

2

Oligonucleotides

Targets Recommended:
Cat. No. Product Name Target Research Areas Chemical Structure
  • HY-145725A
    Baliforsen

    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-177564
    Delpacibart zotadirsen

    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-177454
    Basivarsen linker

    		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-122970
    1,2-Dihydrotanshinone

    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-174226
    RNA binder 2

    		DNA/RNA Synthesis Others
    RNA binder 2 (Compound 20) is a covalent inhibitor targeting the r(CUG)exp RNA in myotonic dystrophy type 1 (DM1). RNA binder 2 binds to RNA via a Hoechst scaffold, blocking MBNL1 binding and restoring normal splicing. RNA binder 2 is promising for research of DM1 .
    RNA binder 2
  • HY-147262
    Etedesiran

    		Antibody-Oligonucleotide Conjugates (AOCs) Small Interfering RNA (siRNA) Neurological Disease
    Etedesiran is a component of the AOC drug Delpacibart etedesiran (HY-177565), formed by the reaction of an siRNA that induces cleavage of mRNA encoding myotonic dystrophy protein kinase (MTPK or DMPK) with SMCC linker (HY-42360). Etedesiran carries a maleimide group at its terminus, which can react with cysteine or lysine and is used for the synthesis of AOC drugs. Etedesiran is applicable to research related to myotonic dystrophy type 1 .
    Etedesiran
  • HY-114567
    GW780056X

    		CDK Neurological Disease Metabolic Disease
    GW780056X is a CDK12 inhibitor. GW780056X decreases nuclear foci count in DM1 cells. GW780056X can be used for the research of myotonic dystrophy type 1 .
    GW780056X

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