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Safety Information

SML4094

RECTAS

new

≥98% (HPLC)

Synonym(s):

2-Chloro-6-Furfurylaminopurine, 2-Chloro-N-(2-furanylmethyl)-7H-purin-6-amine, 2-Chloro-N-(2-furanylmethyl)-9H-purin-6-amine, 2-Chloro-N-[(furan-2-yl)methyl]-7H-purin-6-amine, 2-Chloro-N6-furfuryl-adenine, NSC 45794, NSC-45794, NSC45794

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About This Item

Empirical Formula (Hill Notation):
C10H8ClN5O
CAS Number:
Molecular Weight:
249.66
MDL number:
UNSPSC Code:
12352200

Quality Level

Assay

≥98% (HPLC)

form

powder

color

white to beige

solubility

DMSO: 2 mg/mL, clear (Warmed)

storage temp.

-10 to -25°C

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

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.

Pictograms

Skull and crossbones

Signal Word

Danger

Hazard Statements

Hazard Classifications

Acute Tox. 3 Oral

Storage Class Code

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)
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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