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Givosiran is a small interfering RNA that targets hepatic aminofibrillar synthase 1 (ALAS1) messenger RNA, down-regulates ALAS1 mRNA, and prevents the accumulation of neurotoxic α-aminofibrillar acids and porphyrins, which can be used in porphyria-related studies.
| Molecular Weight | 16300.6 |
| CAS Number | 1639325-43-1 |
| Form | Solid |
| Storage | -20°C, protect from light |
[2] Yahiya Y Syed. Drugs. Givosiran: A Review in Acute Hepatic Porphyria
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