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RECTAS-2.0
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Catalog No. T206725Cas No. 3038234-95-3
RECTAS-2.0 is a small molecule designed to correct RNA mis-splicing caused by the GLA c.639+919G>A mutation, intended for research in Fabry disease.
RECTAS-2.0
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Catalog No. T206725Cas No. 3038234-95-3
RECTAS-2.0 is a small molecule designed to correct RNA mis-splicing caused by the GLA c.639+919G>A mutation, intended for research in Fabry disease.
| Pack Size | Price | USA Warehouse | Global Warehouse | Quantity |
|---|---|---|---|---|
| 10 mg | Inquiry | 10-14 weeks | 10-14 weeks | |
| 50 mg | Inquiry | 10-14 weeks | 10-14 weeks |
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In Stock Estimated shipping dateUSA Warehouse[1-2 days] Global Warehouse[5-7 days]
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Product Introduction
Bioactivity
Chemical Properties
Storage & Solubility Information
| Description | RECTAS-2.0 is a small molecule designed to correct RNA mis-splicing caused by the GLA c.639+919G>A mutation, intended for research in Fabry disease. |
| In vitro | RECTAS-2.0 is capable of restoring normal GLA mRNA splicing and α-galactosidase activity in patient-derived B lymphoblastoid cell lines and cardiomyocytes derived from induced pluripotent stem cells. Furthermore, RECTAS-2.0 shows potential applicability in other genetic diseases involving similar exon competition. |
| Molecular Weight | 388.805 |
| Formula | C18H17ClN4O4 |
| Cas No. | 3038234-95-3 |
| Smiles | N(CC1=CC=CO1)C=2C3=C(C(OC[C@H]4CN(C)C(=O)O4)=NC=C3)N=C(Cl)C2 |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice/Shipping at ambient temperature. |
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In Vivo Formulation Calculator (Clear solution)
Please enter your animal experiment information in the following box and click Calculate to obtain the stock solution preparation method and in vivo formula preparation method:
For example, if the intended dosage is 10 mg/kg for animals weighing 20 g , with a dosing volume of 100 μL per animal, and a total of 10 animals are to be administered, using a formulation of 
10% DMSO+ 40% PEG300+ 5% Tween 80+ 45% Saline/PBS/ddH2O , the resulting working solution concentration would be 2 mg/mL.Stock Solution Preparation:
Dissolve 2 mg of the compound in 100 μL DMSO to obtain a stock solution at a concentration of 20 mg/mL . If the required concentration exceeds the compound's known solubility, please contact us for technical support before proceeding.
Preparation of the In Vivo Formulation:
1) Add 100 μL of the DMSO stock solution to 400 μL PEG300 and mix thoroughly until the solution becomes clear.
2) Add 50 μL Tween 80 and mix well until fully clarified.
3) Add 450 μL Saline,PBS or ddH2O and mix thoroughly until a homogeneous solution is obtained.
This example is provided solely to demonstrate the use of the In Vivo Formulation Calculator and does not constitute a recommended formulation for any specific compound. Please select an appropriate dissolution and formulation strategy based on your experimental model and route of administration.
Dose Conversion
You can also refer to dose conversion for different animals. More Dose Conversion
Tech Support
Please see Inhibitor Handling Instructions for more frequently ask questions. Topics include: how to prepare stock solutions, how to store products, and cautions on cell-based assays & animal experiments, etc
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