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[1].
Product Specifications
CAS Number
[3038234-95-3]
UNSPSC
12352005
Target
DNA/RNA Synthesis
Related Pathways
Cell Cycle/DNA Damage
Field of Research
Metabolic Disease
Smiles
ClC1=NC(C(OC[C@H]2CN(C)C(O2)=O)=NC=C3)=C3C(NCC4=CC=CO4)=C1
Molecular Formula
C18H17ClN4O4
Molecular Weight
388.80
References & Citations
[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.
Shipping Conditions
Room temperature
Scientific Category
Reference compound1
Clinical Information
No Development Reported
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