Nusinersen |
رقم الكتالوجGC64293
One-Click Copy Product Info
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Nusinersen هو دواء قليل النوكليوتيد مضاد للحساسية يعدل تضفير الحمض النووي الريبي قبل المرسال لجين SMN2 وبالتالي يعزز زيادة إنتاج بروتين SMN كامل الطول
Products are for research use only. Not for human use. We do not sell to patients.
Cas No.: 1258984-36-9
Sample solution is provided at 25 µL, 10mM.
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Nusinersen is an SMN-ASO that increases SMN levels by blocking an intronic splice silencer in SMN2, thereby facilitating the exon 7 inclusion and generation of FL-SMN2 transcripts[1].
Nusinersen(6.5mg/kg; I.C.V.) robustly rescues the growth curve of SMN∆7 mice, enhances motor function and dramatically reduces αMN loss and prevents the disruption and loss of canonical Cajal bodies (CBs) in αMNs of the SMA mouse. Also, Nusinersen treatment normalizes the distribution of the protein synthesis machinery and polyadenylated mRNAs in SMNΔ7 mice[2]. Nusinersen was administered to rabbits as an intrathecally (IT) bolus dose of 1.8 mg/kg to the first group, intravenously (IV) bolus dose of 1 mg/kg to the second group, respectively. The Cmax, AUClast and C0 of IV were 15.6±4.88μg/mL, 17.8±4.10 h*μg/mL and 17.6±6.20μg/mL; the Tmax, Cmax and AUClast of IT were 1h, 6.0±0.10μg/mL and 12.4±1.15 h*μg/mL, respectively. The calculated bioavailability of nusinersen was 33 %[3].
References:
[1] Torres-Benito L, Schneider S, Rombo R, et al. NCALD antisense oligonucleotide therapy in addition to nusinersen further ameliorates spinal muscular atrophy in mice[J]. The American Journal of Human Genetics, 2019, 105(1): 221-230.
[2] Berciano M T, Puente-Bedia A, Medina-Samamé A, et al. Nusinersen ameliorates motor function and prevents motoneuron Cajal body disassembly and abnormal poly (A) RNA distribution in a SMA mouse model[J]. Scientific reports, 2020, 10(1): 10738.
[3] Zhang X, Sha C, Zhang W, et al. Development and validation of an HILIC/MS/MS method for determination of nusinersen in rabbit plasma[J]. Heliyon, 2024, 10(10).
| Animal experiment [1]: | |
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Animal models |
WT mice and SMN∆7 mice |
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Preparation Method |
To investigate the efects of nusinersen treatment in the αMNs of SMN∆7 mice, we used 32 animals (16 WT mice and 16 SMN∆7 mice) that were divided into four groups. WT and SMN∆7 mice were treated with 10µl of nusinersen at a dose of 6.5mg/kg (nusinersen group), or with 10µl of a 0.9% saline solution (vehicle group). nusinersen or the vehicle was injected at the day of birth (P0) via ICV using a 10-µl syringe and a custom 33-gauge needle. |
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Dosage form |
6.5mg/kg; I.C.V. |
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Applications |
Treatment with nusinersen robustly rescues the growth curve, enhances motor function and dramatically reduces αMN loss. |
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References: |
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| Cas No. | 1258984-36-9 | SDF | |
| Formula | C234H340N61O128P17S17 | M.Wt | 7127.30 |
| الذوبان | Storage | -20°C, stored under nitrogen, away from moisture | |
| General tips | Please select the appropriate solvent to prepare the stock solution according to the
solubility of the product in different solvents; once the solution is prepared, please store it in
separate packages to avoid product failure caused by repeated freezing and thawing.Storage method
and period of the stock solution: When stored at -80°C, please use it within 6 months; when stored
at -20°C, please use it within 1 month. To increase solubility, heat the tube to 37°C and then oscillate in an ultrasonic bath for some time. |
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| Shipping Condition | Evaluation sample solution: shipped with blue ice. All other sizes available: with RT, or with Blue Ice upon request. | ||
| Prepare stock solution | |||
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1 mg | 5 mg | 10 mg |
| 1 mM | 140.3 μL | 701.5 μL | 1.4031 mL |
| 5 mM | 28.1 μL | 140.3 μL | 280.6 μL |
| 10 mM | 14 μL | 70.2 μL | 140.3 μL |
Step 1: Enter information below (Recommended: An additional animal making an allowance for loss during the experiment)
g
μL
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Calculation results:
Working concentration: mg/ml;
Method for preparing DMSO master liquid: mg drug pre-dissolved in μL DMSO ( Master liquid 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 master liquid, next addμL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL saline, mix and clarify.
Method for preparing in vivo formulation: Take μL DMSO master liquid, next add μL Corn oil, mix and clarify.
Note: 1. Please make sure the liquid is clear before adding the next solvent.
2. Be sure to add the solvent(s) in order. You must ensure that the solution obtained, in the previous addition, is a clear solution before proceeding to add the next solvent. Physical methods such as vortex, ultrasound or hot water bath can be used to aid dissolving.
3. All of the above co-solvents are available for purchase on the GlpBio website.
Quality Control & SDS
- View current batch:
- Purity: >98.50% Appearance: A solid
- COA (Certificate of Analysis)
- SDS (Safety Data Sheet)
- Datasheet
Average Rating: 5 (Based on Reviews and 22 reference(s) in Google Scholar.)