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Art. -Nr.: HY-172552

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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.

Nur für Forschungszwecke. Wir verkaufen nicht an Patienten.

CAS. Nr.: 3038234-95-3
Formel: C₁₈H₁₇ClN₄O₄
Molecular Weight:388.80

Speicherung:

Please store the product under the recommended conditions in the Certificate of Analysis.

Data Sheet (271 KB)

RECTAS-2.0 Chemical Structure

CAS. Nr.: 3038234-95-3

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Preis

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Use of MCE Products

Strong Fluorescence Signal / Multi-Disciplinary Applications / High Fluorescence Specificity

Biologische Aktivität
- Beschreibung
- In Vitro
Chemical Information
Protokoll (1)
Reinheit & Dokumentation
Verweise
Calculators
Help & FAQs
Classifications
Bioz

Biologische Aktivität

Beschreibung

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^[1].

Alle DNA/RNA Synthesis Isoform-spezifische Produkte anzeigen :

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

CAS. Nr. 3038234-95-3
Molecular Weight 388.80
Formel C₁₈H₁₇ClN₄O₄
SMILES

ClC1=NC(C(OC[C@H]2CN(C)C(O2)=O)=NC=C3)=C3C(NCC4=CC=CO4)=C1
Versand

Room temperature in continental US; may vary elsewhere.
Speicherung

Please store the product under the recommended conditions in the Certificate of Analysis.

Protokoll

All Animals and Diseases

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.

Reinheit & Dokumentation

Select Batch:

Reinheit: 99.98%

Assay: 99.98%

ee.: 99.98%

Data Sheet (271 KB)
COA (2659 KB)
Elemental Analysis Report (2659 KB)
HNMR (2659 KB)
CNMR (2659 KB)
RP-HPLC (2659 KB)
NP-HPLC (2659 KB)
MS (2659 KB)
LCMS (2659 KB)
IR (2659 KB)
GC (2659 KB)
OR (2659 KB)
SDS-PAGE (2659 KB)
SEC-HPLC (2659 KB)
Other Report (2659 KB)
Handling Instructions (2659 KB)

Verweise

[1]. Tomonari Awaya, et al. Invention of an oral medication for cardiac Fabry disease caused by RNA mis-splicing. Sci Adv. 2025 Apr 11;11(15):eadt9695. [Content Brief]

Calculators

Molaritätsrechner

Mass (g) = Concentration (mol/L) × Volume (L) × Molecular Weight (g/mol)

Mass Mass

Konzentration Konzentration

Volumen Volumen

Molecular Weight Molecular Weight

Verdünnungsrechner

Konzentration _{(Stammlösung)} × Volumen _{(Stammlösung)} = Konzentration _{(Ziellösung)} × Volumen _{(Ziellösung)}

Konzentration _{(Stammlösung)} Konzentration _{(Stammlösung)}

Volumen _{(Stammlösung)} Volumen _{(Stammlösung)}

Konzentration _{(Ziellösung)} Konzentration _{(Ziellösung)}

Volumen _{(Ziellösung)} Volumen _{(Ziellösung)}

Help & FAQs

How should lyophilized recombinant proteins be reconstituted and stored?

1. Before opening the cap, centrifuge the vial at 13000 rpm for 20-30 seconds. This step will ensure that any lyophilized powder that may have adhered to the cap or walls is collected at the bottom of the vial, minimizing the risk of product loss.

Bioz

Keywords

MOQ

Minimum order quantity

100 mg

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