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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

RECTAS-2.0

😃Good
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 SizePriceAvailabilityQuantity
10 mgInquiry10-14 weeks
50 mgInquiry10-14 weeks
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All TargetMol products are for research purposes only and cannot be used for human consumption. We do not provide products or services to individuals. Please comply with the intended use and do not use TargetMol products for any other purpose.
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Product Introduction

Bioactivity
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.
Chemical Properties
Molecular Weight388.805
FormulaC18H17ClN4O4
Cas No.3038234-95-3
Storage & Solubility Information
StoragePowder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice/Shipping at ambient temperature.

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Mother liquor preparation method: 2 mg of drug dissolved in 50 μL DMSOTargetMol | reagent (mother liquor concentration of 40 mg/mL), if you need to configure a concentration that exceeds the solubility of the product, please contact us first.
Preparation method for in vivo formula: Take 50 μL DMSOTargetMol | reagent main solution, add 300 μLPEG300TargetMol | reagent mix well and clarify, then add 50 more μL Tween 80, mix well and clarify, then add 600 more μLSaline/PBS/ddH2OTargetMol | reagent mix well and clarify
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