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About This Item
Empirical Formula (Hill Notation):
C10H8ClN5O
CAS Number:
Molecular Weight:
249.66
MDL number:
NACRES:
NA.21
Product Name
RECTAS, ≥98% (HPLC)
SMILES string
Clc1nc2[nH]cnc2c(n1)NCc3[o]ccc3
InChI
1S/C10H8ClN5O/c11-10-15-8(7-9(16-10)14-5-13-7)12-4-6-2-1-3-17-6/h1-3,5H,4H2,(H2,12,13,14,15,16)
InChI key
QGUOPVCOXHVPJX-UHFFFAOYSA-N
assay
≥98% (HPLC)
form
powder
color
white to beige
solubility
DMSO: 2 mg/mL, clear (Warmed)
storage temp.
-10 to -25°C
Quality Level
Biochem/physiol Actions
Orally active, CDC2-like kinase 1 (CLK1) allosteric activator that enhances promotes CLK1/SRSF5 pathway-mediated pre-mRNA splicing events.
RECTAS is an orally active, CDC2-like kinase 1 (CLK1) allosteric activator that enhances SRSF6 c-terminal RS domain phosphorylation by CLK1, thereby promoting CLK1/SRSF5 pathway-mediated pre-mRNA splicing events. RECTAS restores IKBKAP-familial dysautonomia (FD) exon 20 inclusion in cultured cells (2-10 μM; FD patient iPSC-SNs & murine neuro 2A cells) and in IKBKAP-FD transgenic mice in vivo (two p.o. doses of 300 or 400 mg/kg 4h apart). RECTAS suppresses MC38 tumor growth (by 37/47% at 10/25 μM at the end of 33-day treatment via daily intratumoral injection) by reducing aberrant splicing events and enhancing the splice-neoantigens production.
RECTAS is an orally active, CDC2-like kinase 1 (CLK1) allosteric activator that enhances SRSF6 c-terminal RS domain phosphorylation by CLK1, thereby promoting CLK1/SRSF5 pathway-mediated pre-mRNA splicing events. RECTAS restores IKBKAP-familial dysautonomia (FD) exon 20 inclusion in cultured cells (2-10 μM; FD patient iPSC-SNs & murine neuro 2A cells) and in IKBKAP-FD transgenic mice in vivo (two p.o. doses of 300 or 400 mg/kg 4h apart). RECTAS suppresses MC38 tumor growth (by 37/47% at 10/25 μM at the end of 33-day treatment via daily intratumoral injection) by reducing aberrant splicing events and enhancing the splice-neoantigens production.
signalword
Danger
hcodes
Hazard Classifications
Acute Tox. 3 Oral
Storage Class
6.1C - Combustible acute toxic Cat.3 / toxic compounds or compounds which causing chronic effects
wgk
WGK 3
flash_point_f
Not applicable
flash_point_c
Not applicable
Regulatory Information
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Shingo Matsushima et al.
Science translational medicine, 14(673), eabn6056-eabn6056 (2022-12-01)
Neoantigen production is a determinant of cancer immunotherapy. However, the expansion of neoantigen abundance for cancer therapeutics is technically challenging. Here, we report that the synthetic compound RECTAS can induce the production of splice-neoantigens that could be used to boost
Specificity, synergy, and mechanisms of splice-modifying drugs
Nature Communications, 15(1), 1880-1880 (2024)
Therapeutic manipulation of IKBKAP mis-splicing with a small molecule to cure familial dysautonomia.
Masahiko Ajiro et al.
Nature communications, 12(1), 4507-4507 (2021-07-25)
Approximately half of genetic disease-associated mutations cause aberrant splicing. However, a widely applicable therapeutic strategy to splicing diseases is yet to be developed. Here, we analyze the mechanism whereby IKBKAP-familial dysautonomia (FD) exon 20 inclusion is specifically promoted by a
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