Nusinersen |
Catalog No.GC64293
One-Click Copy Product Info
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Nusinersen es un SMN-ASO que aumenta los niveles de SMN al bloquear un silenciador de empalme intrónico en SMN2, facilitando así la inclusión del exón 7 y la generación de transcripciones FL-SMN2
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 es un SMN-ASO que aumenta los niveles de SMN al bloquear un silenciador de empalme intrónico en SMN2, facilitando así la inclusión del exón 7 y la generación de transcripciones FL-SMN2[1].
Nusinersen(6.5mg/kg; I.C.V.) rescata de forma robusta la curva de crecimiento de ratones SMN∆7, mejora la función motora y reduce drásticamente la pérdida de αMN y previene la interrupción y pérdida de cuerpos canónicos de Cajal (CBs) en αMNs del ratón SMA. Además, el tratamiento con Nusinersen normaliza la distribución de la maquinaria de síntesis de proteínas y los mRNAs poliadenilados en ratones SMNΔ7[2]. Nusinersen se administró a conejos como una dosis de bolo intratecal (IT) de 1.8 mg/kg al primer grupo, por vía intravenosa (IV) dosis en bolo de 1 mg/kg al segundo grupo, respectivamente. El Cmax, AUClast y C0 de IV fueron 15.6±4.88μg/mL, 17.8±4.10 h*μg/mL y 17.6±6.20μg/mL; el Tmax, Cmax y AUClast de IT fueron 1h, 6.0±0.10μg/mL y 12.4±1.15 h*μg/mL, respectivamente. La biodisponibilidad calculada de nusinersen fue del 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).
| Experimentos con animales [1]: | |
Modelos animales | Ratones WT y ratones SMN∆7 |
Método de preparación | Para investigar los efectos del tratamiento con nusinersen en los αMNs de ratones SMN∆7, utilizamos 32 animales (16 ratones WT mice y 16 ratones SMN∆7) que se dividieron en cuatro grupos. Los ratones WT y SMN∆7 fueron tratados con 10µl de nusinersen a una dosis de 6.5mg/kg (grupo de nusinersen), o con 10µl de una solución salina al 0.9% (grupo de vehículo). nusinersen o el vehículo se inyectó el día del nacimiento (P0) a través de ICV utilizando una jeringa de 10-µl y una aguja personalizada de calibre 33. |
Forma de dosificación | 6.5mg/kg; I.C.V. |
Áreas de aplicación | El tratamiento con nusinersen rescata de forma robusta la curva de crecimiento, mejora la función motora y reduce drásticamente la pérdida de αMN. |
References: | |
| Cas No. | 1258984-36-9 | SDF | |
| Formula | C234H340N61O128P17S17 | M.Wt | 7127.30 |
| Solubility | 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 | |||
|
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
Step 2: Enter the in vivo formulation (This is only the calculator, not formulation. Please contact us first if there is no in vivo formulation at the solubility Section.)
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.)