1. Epigenetics
  2. MicroRNA
  3. Farabursen

Farabursen  (Synonyms: RGLS8429; RG1015)

Cat. No.: HY-150364 Purity: 97.59%
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Farabursen (RGLS8429; RG1015) is a blood-brain barrier-permeable miR-17 inhibitor. Farabursen derepresses Pkd1 and Pkd2, the target genes of miR-17, increases the levels of PC1 and PC2, and reduces cyst growth. Farabursen decreases renal cyst growth, kidney weight-to-body weight ratio, cyst index, proliferation index, and blood urea nitrogen levels in mouse models. Farabursen is applicable to research related to autosomal dominant polycystic kidney disease.

For research use only. We do not sell to patients.

RNA, (P-thio)([2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]A-(3′→4′)-[2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]G-Cm-(2′-deoxy-2′-fluoro)A-(2′-deoxy-2′-fluoro)C-(2′-deoxy-2′-fluoro)U-Um-(3′→4′)-[2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]U-(3′→4′)-[2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]A)

Farabursen Chemical Structure

CAS No. : 2953012-32-1

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Description

Farabursen (RGLS8429; RG1015) is a blood-brain barrier-permeable miR-17 inhibitor. Farabursen derepresses Pkd1 and Pkd2, the target genes of miR-17, increases the levels of PC1 and PC2, and reduces cyst growth. Farabursen decreases renal cyst growth, kidney weight-to-body weight ratio, cyst index, proliferation index, and blood urea nitrogen levels in mouse models. Farabursen is applicable to research related to autosomal dominant polycystic kidney disease[1][2].

In Vitro

Farabursen (0-3 μM; 30 s preincubation) exhibits only weak inhibitory activity against human GluA1/GluA4 AMPAR in HEK293 cells, with an inhibition rate of approximately 40% at the concentration of 3 μM[2].
Farabursen (0.1-100 nM; 24 h) potently inhibits the activity of miR-17 in HeLa cells with an EC50 of 22.4 nM, relieves the expression inhibition of miR-17 target gene PKD1 with an EC50 of 43.54 nM, and relieves the expression inhibition of miR-17 target gene PKD2 with an EC50 of 40.76 nM[2].
Farabursen (300 nM; 24-72 h) derepresses the expression of *Pkd1* and *Pkd2* genes, and increases the protein levels of PC1 and PC2 in mouse Pkd1RC/- ADPKD cells[2].
Farabursen (120 nM; 72 h post-transfection) increases the protein levels of PC1 and PC2, and inhibits cyst growth in primary human ADPKD cyst cells with the PKD1+/02641 genotype[2].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Western Blot Analysis[2]

Cell Line: Mouse Pkd1^RC/- ADPKD cells
Concentration: 300 nM
Incubation Time: 24 h post-transfection (gene expression analysis); 72 h post-transfection (protein levels analysis)
Result: Significantly de-repressed Pkd1 and Pkd2 gene expression with fold changes of ~0.6760 and ~0.9286, respectively.
Increased PC1 and PC2 protein levels.
In Vivo

Farabursen (30 mg/kg; subcutaneous injection; single dose) exhibits kidney-preferential distribution and sustained target binding in C57BL/6 mice, with a renal EC75 of 2.2 μg/g[2].
Farabursen (20 mg/kg; subcutaneous injection; postnatal days 8-15) reduces cyst growth, kidney enlargement, cell proliferation, and renal function decline in Pkd1F/RC ADPKD mice[2].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Animal Model: C57BL/6[2]
Dosage: 30 mg/kg
Administration: s.c.; single dose
Result: Showed a pharmacokinetic profile characterized by rapid absorption, rapid clearance, and preferential distribution to the kidney (kidney AUC_last exceeded liver AUC_last by >20-fold).
Reached ≥75% target engagement (percent inhibition of miR-17) in kidney tissue, which was maintained for 14 days; target engagement in liver tissue was ≤75% by 7 days.
Achieved a kidney EC75 (concentration corresponding to 75% miR-17 inhibition) of 2.2 μg/g.
Animal Model: Ksp/Cre; Pkd1F/RC (autosomal dominant polycystic kidney disease model)[2]
Dosage: 20 mg/kg
Administration: s.c.; on postnatal days 8, 10, 12, 15
Result: Reduced kidney weight-to-body weight ratio, cyst index, proliferation index, and blood urea nitrogen (BUN) levels.
Molecular Weight

3066.36

CAS No.
Appearance

Solid

Color

White to off-white

SMILES

[Farabursen]

Shipping

Room temperature in continental US; may vary elsewhere.

Storage

-20°C, sealed storage, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

Purity & Documentation

Purity: 97.59%

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    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

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