RECTAS
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Description: IKBKAP pre-mRNA splicing modulator
Chemical Name: 2-Chloro-N-(2-furanylmethyl)-7H-purin-6-amine
Purity: ≥98%
Biological Activity
RECTAS (rectifier of aberrant splicing) is a pre-mRNA splicing modulator of the IKBKAP (inhibitor of κ light polypeptide gene enhancer in B-cells, kinase complex associated protein) gene. It corrects aberrant IKBKAP splicing in vitro in cellular models of familial dysautonomia and in vivo in transgenic mice. In combination with Phenylbutyric acid (Cat. No. 2682), RECTAS rescues PARK7 aberrant splicing and neuronal cell loss in a midbrain organoid model of familial Parkinson's disease. Orally bioavailable and brain penetrant.Technical Data
M.Wt:
249.66
Formula:
C10H8ClN5O
Solubility:
Soluble to 100 mM in DMSO
Purity:
≥98%
Storage:
Store at -20°C
CAS No:
101862-47-9
The technical data provided above is for guidance only.
For batch specific data refer to the Certificate of Analysis.
Tocris products are intended for laboratory research use only, unless stated otherwise.
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