RECTAS | pre-mRNA splicing modulator
Available Options
| Size: | Price | Quantity | |
|---|---|---|---|
| 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:
- Yoshida et al. (2015), Rectifier of aberrant mRNA splicing recovers tRNA modification in familial dysautonomia; Proc. Natl. Acad. Sci. USA 112 2764
- Ajiro et al. (2021), Therapeutic manipulation of IKBKAP mis-splicing with a small molecule to cure familial dysautonomia; Nat. Commun. 12 4507
- Boussaad et al. (2020), A patient-based model of RNA mis-splicing uncovers treatment targets in Parkinson’s disease; Sci. Transl. Med. 12 eaau3960
C10H8ClN5O
>98% TLC
NMR: (Conforms)
Safety Data Sheet:
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
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:
- Yoshida et al. (2015), Rectifier of aberrant mRNA splicing recovers tRNA modification in familial dysautonomia; Proc. Natl. Acad. Sci. USA 112 2764
- Ajiro et al. (2021), Therapeutic manipulation of IKBKAP mis-splicing with a small molecule to cure familial dysautonomia; Nat. Commun. 12 4507
- Boussaad et al. (2020), A patient-based model of RNA mis-splicing uncovers treatment targets in Parkinson’s disease; Sci. Transl. Med. 12 eaau3960
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