RECTAS-2.0
RECTAS-2.0 is a small molecule designed to correct RNA mis-splicing caused by the GLA c.639+919G>A mutation, used for the research of Fabry disease.
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- CAS No.: 3038234-95-3
- Formule: C18H17ClN4O4
- Masse moléculaire:388.80
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Stockage:
Please store the product under the recommended conditions in the Certificate of Analysis.
To place orders, for customer services and technical support, please contact: MedChemExpress USA
Tel: 609-228-6898 E-mail: [email protected] [email protected]
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Activité biologique
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Chemical Information
- Protocole
- Pureté et documentation
- Références
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Help & FAQs
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Activité biologique
Description
In Vitro
RECTAS-2.0 restores normal GLA mRNA splicing and α-galactosidase activity in patient-derived B-lymphoblastoid cell lines and induced pluripotent stem cell-derived cardiomyocytes[1].
RECTAS-2.0 demonstrates potential applicability to other genetic disorders that involve similar exon competition[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only. Further protocols information, click here.
Chemical Information
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CAS No. 3038234-95-3
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Masse moléculaire 388.80
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Formule C18H17ClN4O4
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SMILES
ClC1=NC(C(OC[C@H]2CN(C)C(O2)=O)=NC=C3)=C3C(NCC4=CC=CO4)=C1
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Livraison
Room temperature in continental US; may vary elsewhere.
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Stockage
Please store the product under the recommended conditions in the Certificate of Analysis.
Protocole
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Genotoxicity/Mutagenicity Study
The bacterial reverse mutation assay detects point mutations that restore amino-acid prototrophy in auxotrophic Salmonella typhimurium or Escherichia coli tester strains; after exposure to a test article, mutagenic activity is read out as an increased number of revertant colonies on minimal agar compared with the vehicle control. The assay uses tester strains with different mutation targets so that base-substitution and frameshift mutagens can be detected, and testing is performed with and without exogenous mammalian metabolic activation because some chemicals require biotransformation to become mutagenic.
Pureté et documentation
Références
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
RECTAS-2.0 Related Classifications
HY-172552 Related Classifications
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