Givosiran (ALN-AS1) is a small interfering RNA compound that selectively targets hepatic aminolevulinate synthase 1 (ALAS1) mRNA, leading to efficient downregulation of ALAS1 expression and consequent inhibition of neurotoxic delta-aminolevulinic acid and porphobilinogen accumulation. Givosiran provides a mechanism-driven research tool with significant relevance for studying metabolic dysregulation and therapeutic intervention strategies in acute intermittent porphyria.
Molekulargewicht:
16300.6
Reinheit:
99.88%
CAS Nummer:
[1639325-43-1]
Target-Kategorie:
Others
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