Nusinersen |
カタログ番号GC64293
One-Click Copy Product Info
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NusinersenはSMN-ASOであり、SMN2でイントロニックスプライスサイレンサーを遮断することでSMNレベルを増やし、よってエクソン7の包含とFL-SMN2の生成を促進する。
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はSMN-ASOであり、SMN2でイントロニックスプライスサイレンサーを遮断することでSMNレベルを増やし、よってエクソン7の包含とFL-SMN2の生成を促進する[1]。
Nusinersen(6.5mg/kg；I.C.V.)はSMN∆7マウスの成長曲線を著しく救援でき、運動機能を増強させ、αMNの喪失を劇的に減らし、SMAマウスのαMNで、正統なカハール体（CB）の崩壊と喪失を阻止する。または、Nusinersenの処理は、SMNΔ7マウスで、タンパク質合成機構とポリアデニル化mRNAの分布を正常化する[2]。グループ1のウサギに対して、Nusinersenを1.8mg/kgの用量で、脊髄内（IT）ボーラス投与をし、グループ2には1mg/kgの用量で、静脈内（IV）ボーラス投与をした。IVのCmax、AUClastとC0は15.6±4.88μg/mL、17.8±4.10 h*μg/mLと17.6±6.20μg/mL；ITのTmax、CmaxとAUClastはそれぞれ1h、6.0±0.10μg/mLと12.4±1.15 h*μg/mLである。nusinersenの計算された生物利用度は33%である[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).
| 動物実験 [1]: | |
動物モデル | WTマウスとSMN∆7マウス |
準備方法 | SMN∆7マウスのαMNsにおけるnusinersen処理の効果を調査するため、4つのグループに分けた32匹の動物（16 WT匹のマウスと16匹のSMN∆7マウス）を使った。WTとSMN∆7マウスに、用量が6.5mg/kgの10µlのnusinersen（nusinersenグループ）、または10μlの0.9%の生理食塩水（溶媒グループ）を投与した。10μlシリンジとカスタム33ゲージ針を使い、出生当日（P0）に、nusinersenまたは溶媒をICVで注射した。 |
投与形態 | 6.5mg/kg；I.C.V. |
アプリケーション | Nusinersenの処理はSMN∆7マウスの成長曲線を著しく救援でき、運動機能を増強させ、αMNの喪失を劇的に減らした。 |
References: | |
| Cas No. | 1258984-36-9 | SDF | |
| Formula | C234H340N61O128P17S17 | M.Wt | 7127.30 |
| 溶解度 | 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.)