Nusinersen |
Catalog No.GC64293
One-Click Copy Product Info
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Nusinersen은 SMN2의 플러그 머플러를 차단하고 SMN 수준을 증가시키고 exon 7의 포함과 FL-SMN2 전사체의 생성을 촉진하는 SMN-ASO이다.
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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Nusinersen은 SMN2의 플러그 머플러를 차단하고 SMN 수준을 증가시키고 exon 7의 포함과 FL-SMN2 전사체의 생성을 촉진하는 SMN-ASO이다.
Nusinersen (6.5 mg/kg; I.C.V.)은 SMN∆7 쥐의 성장 곡선을 강력하게 구출하고 운동 기능을 향상시키고 αMN 손실을 현저하게 줄이고 SMA 쥐의 αMNs에서 전형적인 Cajal bodies (CBs)의 파괴와 손실을 방지한다. 게다가 Nusinersen 치료는 SMNΔ7 쥐에서 단백질 합성 기계와 polyadenylated mRNAs의 분포를 정상화시킨다[2].Nusinersen은 토끼에게 첫 번째 그룹에는 1.8 mg/kg의 경막하 (IT) 단일 투여량으로 두 번째 그룹에는 1 mg/kg의 정맥 (IV) 단일 투여량으로 각각 투여되었다. IV의 Cmax, AUClast, C0는 각각 15.6±4.88μg/mL, 17.8±4.10 hμg/mL, 17.6±6.20μg/mL였다. IT의 Tmax, Cmax, AUClast는 각각 1시간, 6.0±0.10μg/mL, 12.4±1.15 hμg/mL이었다. Nusinersen의 계산된 생체이용률은 33%였다[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).
| 동물 실험 [1]: | |
동물 모형 | WT 쥐 및 SMN∆7 쥐 |
제조 방법 | SMN∆7 쥐의 αMNs에서 Nusinersen 치료의 효과를 조사하기 위해 32 마리의 동물 (16 마리의 WT 쥐와 16 마리의 SMN∆7 쥐)을 사용하고 네 그룹으로 나누었습니다. WT과 SMN∆7 쥐는 6.5mg/kg의 농도로 10µl의 Nusinersen (Nusinersen 그룹) 또는 0.9% 염수 10µl (차량 그룹)을 투여받았습니다. Nusinersen 또는 차량은 10-µl 주사기와 맞춤 33-gauge 바늘을 사용하고 출생일 (P0)에 ICV를 통해 주사되었습니다. |
제형 | 6.5mg/kg; I.C.V. |
응용 분야 | Nusinersen으로 치료하면 성장 곡선을 강력하게 구출하고 운동 기능을 향상시키고 αMN 손실을 현저하게 줄일 수 있습니다. |
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 | |||
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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
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.)