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Eplontersen is a triantennary N-acetylgalactosamine–conjugated antisense oligonucleotide that selectively inhibits both wild-type and mutant transthyretin mRNA, thereby reducing pathogenic TTR protein production and amyloid fibril formation associated with ATTR amyloidosis affecting cardiac and peripheral nerve tissues.
| Pack Size | Price | USA Warehouse | Global Warehouse | Quantity |
|---|---|---|---|---|
| 1 mg | $202 | 2-4 weeks | 2-4 weeks | |
| 5 mg | $585 | 2-4 weeks | 2-4 weeks | |
| 10 mg | $978 | 2-4 weeks | 2-4 weeks | |
| 50 mg | Inquiry | 2-4 weeks | 2-4 weeks |
| Description | Eplontersen is a triantennary N-acetylgalactosamine–conjugated antisense oligonucleotide that selectively inhibits both wild-type and mutant transthyretin mRNA, thereby reducing pathogenic TTR protein production and amyloid fibril formation associated with ATTR amyloidosis affecting cardiac and peripheral nerve tissues. |
| In vivo | In in vivo studies using transgenic C57BL/6 mice expressing human transthyretin (hTTR), Eplontersen exhibits dose-dependent gene silencing activity. Following subcutaneous (s.c.) administration at doses of 0.6, 2, and 6 mg/kg (once weekly for 3 weeks), the substance results in a reduction of TTR expression. In the 6 mg/kg dose group, hepatic TTR mRNA levels were observed to decrease to 15% of baseline, while plasma TTR protein levels were reduced to 21% by day 17 [3]. |
| Cas No. | 1637600-16-8 |
| Sequence | DNA, d([2′-O-(2-methoxyethyl)]m5rU-sp-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]rG-G-sp-T-sp-T-sp-A-sp-m5C-sp-A-sp-T-sp-G-sp-A-sp-A-sp-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rC-sp-[2′-O-(2-methoxyethyl)]m5rC-sp-[2′-O-(2-methoxyethyl)]m5rC), 5′-[26-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-14,14-bis[[3-[[6-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]hexyl]amino]-3-oxopropoxy]methyl]-8,12,19-trioxo-16-oxa-7,13,20-triazahexacos-1-yl hydrogen phosphate] |
| Storage | In solvent: -80°C for 1 year | Shipping with blue ice/Shipping at ambient temperature. |
| Solubility Information | H2O: 80 mg/mL, Sonication is recommended. |
Dissolve 2 mg of the compound in 100 μL DMSO
to obtain a stock solution at a concentration of 20 mg/mL . If the required concentration exceeds the compound's known solubility, please contact us for technical support before proceeding.
1) Add 100 μL of the DMSO
stock solution to 400 μL PEG300
and mix thoroughly until the solution becomes clear.
2) Add 50 μL Tween 80 and mix well until fully clarified.
3) Add 450 μL Saline,PBS or ddH2O
and mix thoroughly until a homogeneous solution is obtained.
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