| Size | Price | Stock | Qty |
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| 1mg |
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| Other Sizes |
| Targets |
Target is dystrophin pre-mRNA; specifically, it binds to exon 51 of the dystrophin transcript to induce skipping. It acts on the splicing machinery to restore the reading frame, enabling production of a shorter but partially functional dystrophin protein in DMD patients with amenable mutations. Drisapersen also targets RNase H1 for degradation of the targeted pre-mRNA.
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| ln Vitro |
In vitro studies demonstrate that Drisapersen induces specific exon 51 skipping in human DMD patient myotubes in a dose-dependent manner, resulting in dystrophin protein restoration. The FITC-labeled version shows similar bioactivity while allowing visualization of cellular uptake and nuclear accumulation. FITC conjugation does not interfere with its antisense activity or splicing modulation capability.
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| ln Vivo |
In vivo, Drisapersen administered subcutaneously in mdx mouse models leads to dystrophin restoration in skeletal muscle tissues. The FITC-labeled version exhibits comparable tissue biodistribution, with accumulation observed in muscle, kidney, and liver following systemic administration. Fluorescence imaging enables real-time tracking of oligonucleotide localization and persistence in target tissues.
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| Enzyme Assay |
Standard assay protocol: Incubate FITC-labeled Drisapersen with purified dystrophin pre-mRNA substrate in a cell-free splicing system. After incubation at 37degC for 2-4 hours, extract RNA and perform RT-PCR to assess exon 51 skipping efficiency. Use fluorescent detection to quantify binding affinity and specificity.
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| Cell Assay |
Culture human DMD patient myotubes in differentiation medium. Treat with FITC-labeled Drisapersen at concentrations ranging from 1-20 uM for 24-48 hours. Fix cells with 4% paraformaldehyde, stain nuclei with DAPI, and visualize cellular uptake and localization by fluorescence microscopy or confocal imaging. Quantify exon skipping by RT-PCR and dystrophin protein by Western blot.
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| Animal Protocol |
Administer FITC-labeled Drisapersen to mdx mice via subcutaneous injection at doses of 3-9 mg/kg. Collect tissues (muscle, heart, kidney, liver) at various time points (1, 6, 24, 48, 72 hours post-dose). Process tissues for fluorescence imaging to track oligonucleotide distribution. Extract RNA for exon skipping analysis and protein for dystrophin quantification by Western blot.
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| ADME/Pharmacokinetics |
As a research tool, the compound is used primarily for tracking biodistribution rather than therapeutic PK. Drisapersen itself, upon subcutaneous administration, reaches Cmax within 1-4 hours, has a half-life of approximately 2-4 hours in plasma, and distributes extensively to muscle tissues. Renal clearance is the primary elimination pathway.
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| Toxicity/Toxicokinetics |
A toxicology profile is not fully established for the FITC-labeled form. However, in clinical trials with Drisapersen, adverse effects included injection site reactions, pyrexia, proteinuria, and transient thrombocytopenia. No significant safety concerns were observed at doses of 3 and 6 mg/kg, though the 9 mg/kg dose showed increased adverse events.
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| References |
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| Additional Infomation |
The FITC label enables fluorescent tracking of the oligonucleotide's cellular and tissue distribution. Drisapersen was developed by Prosensa and GSK, and underwent Phase III clinical trials for DMD, though regulatory approval was not ultimately obtained due to concerns over clinical benefit. The compound remains a valuable research tool for studying antisense oligonucleotide-mediated exon skipping.
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| Molecular Weight |
7962.60
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| Appearance |
White to yellow solid powder
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| HS Tariff Code |
2934.99.9001
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| Storage |
Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month Note: Please store this product in a sealed and protected environment, avoid exposure to moisture. |
| Shipping Condition |
Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)
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| Solubility (In Vitro) |
H2O :~25 mg/mL (~3.14 mM)
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| Solubility (In Vivo) |
Note: Listed below are some common formulations that may be used to formulate products with low water solubility (e.g. < 1 mg/mL), you may test these formulations using a minute amount of products to avoid loss of samples.
Injection Formulations
Injection Formulation 1: DMSO : Tween 80： Saline = 10 : 5 : 85 (i.e. 100 μL DMSO stock solution → 50 μL Tween 80 → 850 μL Saline)(e.g. IP/IV/IM/SC) *Preparation of saline: Dissolve 0.9 g of sodium chloride in 100 mL ddH ₂ O to obtain a clear solution. Injection Formulation 2: DMSO : PEG300 ：Tween 80 : Saline = 10 : 40 : 5 : 45 (i.e. 100 μL DMSO → 400 μLPEG300 → 50 μL Tween 80 → 450 μL Saline) Injection Formulation 3: DMSO : Corn oil = 10 : 90 (i.e. 100 μL DMSO → 900 μL Corn oil) Example: Take the Injection Formulation 3 (DMSO : Corn oil = 10 : 90) as an example, if 1 mL of 2.5 mg/mL working solution is to be prepared, you can take 100 μL 25 mg/mL DMSO stock solution and add to 900 μL corn oil, mix well to obtain a clear or suspension solution (2.5 mg/mL, ready for use in animals). View More
Injection Formulation 4: DMSO : 20% SBE-β-CD in saline = 10 : 90 [i.e. 100 μL DMSO → 900 μL (20% SBE-β-CD in saline)] Oral Formulations
Oral Formulation 1: Suspend in 0.5% CMC Na (carboxymethylcellulose sodium) Oral Formulation 2: Suspend in 0.5% Carboxymethyl cellulose Example: Take the Oral Formulation 1 (Suspend in 0.5% CMC Na) as an example, if 100 mL of 2.5 mg/mL working solution is to be prepared, you can first prepare 0.5% CMC Na solution by measuring 0.5 g CMC Na and dissolve it in 100 mL ddH2O to obtain a clear solution; then add 250 mg of the product to 100 mL 0.5% CMC Na solution, to make the suspension solution (2.5 mg/mL, ready for use in animals). View More
Oral Formulation 3: Dissolved in PEG400 (Please use freshly prepared in vivo formulations for optimal results.) |
| Preparing Stock Solutions | 1 mg | 5 mg | 10 mg | |
| 1 mM | 0.1256 mL | 0.6279 mL | 1.2559 mL | |
| 5 mM | 0.0251 mL | 0.1256 mL | 0.2512 mL | |
| 10 mM | 0.0126 mL | 0.0628 mL | 0.1256 mL |
*Note: Please select an appropriate solvent for the preparation of stock solution based on your experiment needs. For most products, DMSO can be used for preparing stock solutions (e.g. 5 mM, 10 mM, or 20 mM concentration); some products with high aqueous solubility may be dissolved in water directly. Solubility information is available at the above Solubility Data section. Once the stock solution is prepared, aliquot it to routine usage volumes and store at -20°C or -80°C. Avoid repeated freeze and thaw cycles.
Calculation results
Working concentration： mg/mL；
Method for preparing DMSO stock solution： mg drug pre-dissolved in μL DMSO (stock solution concentration mg/mL). Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug.
Method for preparing in vivo formulation:：Take μL DMSO stock solution, next add μL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O，mix and clarify.
(1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
(2) Be sure to add the solvent(s) in order.
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