Givosiran (ALN-AS1) sodium is a small interfering RNA that targets hepatic aminolevulinate synthase 1 (ALAS1) messenger RNA. Givosiran sodium downregulates ALAS1 mRNA and prevents accumulation of neurotoxic delta-aminolevulinic acid (ALA) and porphobilinogen (PBG) levels. Givosiran sodium demonstrates potent inhibitory activity against ALAS1 in mouse, rat, and cynomolgus monkey models. Givosiran sodium can be used for the research of acute hepatic porphyria (AHP)[1][2].
Molekulargewicht:
16300.60
Reinheit:
98.71
CAS Nummer:
[1639325-44-2]
Formel:
C524H650F16N173Na44O316P43S6
Target-Kategorie:
Small Interfering RNA (siRNA)
Anwendungsbeschreibung:
Oligonucleotides
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