RECTAS | pre-mRNA splicing modulator

CAS:
 101862-47-9
Catalog Number:
 10-5273

Available Options

Size:PriceQuantity 
5 mg$70.00
25 mg$220.00

RECTAS (101862-47-9), a pre-mRNA splice modulator (Rectifier of Aberrant Splicing), was discovered in a small molecule screen for compounds that rectify aberrant IKBKAP splicing in cells from patients with familial dysautonomia. RECTAS (2 mM) increased the expression of IKAP and restored tRNA modifications.1,2 It directly interacts with CDC-like kinases enhancing SRSF6 phosphorylation.2  A combination treatment with RECTAS and phenylbutyric acid was shown to restore DJ-1 protein and mitochondrial dysfunction in Parkinson’s disease models.3

References/Citations:

  1. Yoshida et al. (2015), Rectifier of aberrant mRNA splicing recovers tRNA modification in familial dysautonomia; Proc. Natl. Acad. Sci. USA 112 2764
  2. Ajiro et al. (2021), Therapeutic manipulation of IKBKAP mis-splicing with a small molecule to cure familial dysautonomia; Nat. Commun. 12 4507
  3. Boussaad et al. (2020), A patient-based model of RNA mis-splicing uncovers treatment targets in Parkinson’s disease; Sci. Transl. Med. 12 eaau3960
CAS:
101862-47-9
Catalog Number:
10-5273
Activity:
pre-mRNA splicing modulator
Chemical Names:
2-Chloro-N-(2-furanylmethyl)-9H-purin-6-amine
Molecular Weight:
249.66
Molecular Formula:

C10H8ClN5O

Solubility:
Soluble in DMSO (27 mg/ml)
Physical Properties:
Pale yellow solid
Purity:

>98% TLC
NMR: (Conforms)

Storage Temperature:
-20°C
Stability:
Stable for 2 years from date of purchase as supplied. Solutions in DMSO may be stored at -20°C for up to 3 months.
Shipping Code:
RT

Safety Data Sheet:

N/A

Product Data Sheet:

Materials provided by Focus Biomolecules are for laboratory research use only and are not intended for human or veterinary applications. Please note that we do not sell to individuals and that all orders placed by non-research organizations will incur a $20 restocking/refund fee