Patisiran is a small double-stranded interfering RNA that targets sequences within the Transthyretin (TTR) messenger RNA and specifically inhibits hepatic synthesis of mutant and wild-type TTR. It can be used to study hereditary TTR amyloidosis.
| CAS Number | 1420706-45-1 |
| Storage |
Powder -20°C 3 years ; 4°C 2 years In solvent -80°C 6 months ; -20°C 1 month |
| Species | Mouse | Rat | Rabbit | Guinea pig | Hamster | Dog |
| Weight (kg) | 0.02 | 0.15 | 1.8 | 0.4 | 0.08 | 10 |
| Body Surface Area (m2) | 0.007 | 0.025 | 0.15 | 0.05 | 0.02 | 0.5 |
| Km factor | 3 | 6 | 12 | 8 | 5 | 20 |
| Animal A (mg/kg) = Animal B (mg/kg) multiplied by | Animal B Km |
| Animal A Km |
For example, to modify the dose of Compound A used for a mouse (20 mg/kg) to a dose based on the BSA for a rat, multiply 20 mg/kg by the Km factor for a mouse and then divide by the Km factor for a rat. This calculation results in a rat equivalent dose for Compound A of 10 mg/kg.
| Related Transthyretin (TTR) Products |
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| Eplontersen
Eplontersen is an N-acetylgalactosamine (GalNAc3)-suffixed antisense oligonucleotide (ASO) that targets the mRNA of the thyroxine transporter protein, TTR, and inhibits both variant and wild-type TTR production. It can be used in studies related to amyloidotic polyneuropathy. |
| WT-TTR inhibitor 1
WT-TTR inhibitor 1 is a wild-type Transthyretin (WT-TTR) inhibitor with 29.05% inhibition at 100 μM. |
| AS-Patisiran
Patisiran is a double-stranded small interfering RNA that targets a sequence within the transthyretin (TTR) messenger RNA. |
| Vutrisiran
Vutrisiran is a liver-directed small interfering RNA (siRNA) that targets transthyretin protein (TTR) mRNA and can be used in TTR-mediated amyloidosis studies. |
| Inotersen
Inotersen is an antisense oligonucleotide that inhibits the production of the hepatic transthyretin (TTR) protein. It can be used in studies related to TTR-mediated amyloidosis. |
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