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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 | Availability | Quantity |
|---|---|---|---|
| 10 mg | Inquiry | 10-14 weeks | |
| 50 mg | Inquiry | 10-14 weeks |
| 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 |
| 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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