Givosiran (ALN-AS1) is a small interfering RNA that targets hepatic aminolevulinate synthase 1 (ALAS1) messenger RNA. Givosiran downregulates ALAS1 mRNA and prevents accumulation of neurotoxic delta-aminolevulinic acid (ALA) and porphobilinogen (PBG) levels. Givosiran demonstrates potent inhibitory activity against ALAS1 in mouse, rat, and cynomolgus monkey models. Givosiran can be used for the research of acute hepatic porphyria (AHP)[1][2].
Molekulargewicht:
16300.60
Reinheit:
98.51
CAS Nummer:
[1639325-43-1]
Formel:
C524H694F16N173O316P43S6
Target-Kategorie:
Small Interfering RNA (siRNA)
Anwendungsbeschreibung:
Oligonucleotides
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