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United States

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Referencia número: 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.

Para uso exclusivo en investigación. No vendemos a pacientes.

No. CAS: 3038234-95-3
Fòrmula: C₁₈H₁₇ClN₄O₄
Peso molecular:388.80

Almacenamiento:

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

Data Sheet (271 KB)

RECTAS-2.0 Chemical Structure

No. CAS: 3038234-95-3

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Use of MCE Products

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

Actividad biológica
- Descripciòn
- In Vitro
Chemical Information
Pureza y Documentación
Referencias
Calculators
Help & FAQs
Classifications
Bioz

Actividad biológica

Descripciòn

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].

Ver todos los productos específicos de isoformas DNA/RNA Synthesis :

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.

Chemical Information

No. CAS 3038234-95-3
Peso molecular 388.80
Fòrmula C₁₈H₁₇ClN₄O₄
SMILES

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

Room temperature in continental US; may vary elsewhere.
Almacenamiento

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

Pureza y Documentación

Select Batch:

Pureza: 99.98%

Assay: 99.98%

ee.: 99.98%

Ficha de datos (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)
Instrucciones de manejo (2659 KB)

Referencias

[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

Calculadora de molaridad

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

Mass Mass

Concentration Concentration

Volume Volume

Molecular Weight Molecular Weight

Calculadora de dilución

Concentration _(start) × Volume _(start) = Concentration _(final) × Volume _(final)

Concentration _(start) Concentration _(start)

Volume _(start) Volume _(start)

Concentration _(final) Concentration _(final)

Volume _(final) Volume _(final)

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

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MOQ

Minimum order quantity

100 mg

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