Unconjugated/naked Etedesiran (without SMCC linker)
Unconjugated/naked Etedesiran (without SMCC linker) is a small interfering RNA (siRNA) targeting DMPK, and also represents the unconjugated, non-SMCC linker-bound component of delpacibart etedesiran. Unconjugated/naked Etedesiran (without SMCC linker) corrects pathogenic mRNA aberrant splicing by targeting and degrading mutant DMPK mRNA, thereby releasing sequestered MBNL splicing factors. Unconjugated/naked Etedesiran (without SMCC linker) can be used in studies of myotonic dystrophy type 1.
For research use only. We do not sell to patients.
- Molecular Weight:13479.92
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Storage:
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
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Biological Activity
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Chemical Information
- Protocols
- Purity & Documentation
- References
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Help & FAQs
All Antibody-Oligonucleotide Conjugates (AOCs) Isoforms
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Biological Activity
Description
IC50 & Target
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AOC Oligonucleotides |
In Vitro
Unconjugated/naked Etedesiran (without SMCC linker) (5 nM; 3 or 4 days) reduces DMPK mRNA by approximately 60% in the nuclear fraction of immortalized DM1 myotubes and by approximately 80% in the cytoplasmic fraction[1].
Unconjugated/naked Etedesiran (without SMCC linker) (1 nM; 7-9 days) reduces CUG repeat nuclear foci by approximately 50% in primary DM1 myotubes[1].
Unconjugated/naked Etedesiran (without SMCC linker) (5 nM; 4 days after induction of differentiation) corrects hundreds of disease-associated aberrant splicing events in immortalized DM1 myotubes, with no effect on splicing in healthy human myotubes[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only. Further protocols information, click here.
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Cell Line:immortalized human myoblasts with myotonic dystrophy type 1 (DM1) and 2,600 CUG repeats
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Concentration:5 nM
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Incubation Time:3 or 4 days
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Result:Reduced DMPK mRNA by approximately 60% in the nuclear fraction relative to mock-treated controls.
Reduced DMPK mRNA by approximately 80% in the cytoplasmic fraction relative to mock-treated controls.
Chemical Information
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Appearance Solid
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Molecular Weight 13479.92
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SMILES
[Unconjugated/naked Etedesiran (without SMCC linker)]
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Shipping
Room temperature in continental US; may vary elsewhere.
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Storage
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Protocols
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RNA extraction experimental
By lysing cells, releasing RNA, and removing impurities such as proteins and DNA, high-purity RNA products are finally obtained. The commonly used traditional method is the guanidine isothiocyanate/phenol/chloroform method (Trizol), which is suitable for a variety of animal materials including animal tissues, microorganisms, cultured cells, etc., and most plant materials.
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RNA interference technology
RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing gene transcription or activating RNA degradation. This mechanism was discovered in plants in 1998 by Andrew Fire and Craig Mello. Today, this phenomenon can be observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals.
Purity & Documentation
References
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
Unconjugated/naked Etedesiran (without SMCC linker) Related Classifications
HY-147262D Related Classifications
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Keywords
- Unconjugated/naked Etedesiran (without SMCC linker)
- Antibody-Oligonucleotide Conjugates (AOCs)
- Small Interfering RNA (siRNA)
- Myotonin-Protein Kinase (DMPK)
- healthy human myotubes
- small interfering RNA
- immortalized DM1 myotubes
- CUG repeat nuclear foci
- siRNA
- immortalized DM1 myoblasts
- primary DM1 myotubes
- DMPK
- Inhibitor
- inhibitor
- inhibit