RNA Interference Therapy Targeting Apolipoprotein C-III in Hypertriglyceridemia.

Daniel Gaudet,Peter Clifton,David Sullivan,John Baker,Christian Schwabe,Susan Thackwray,Russell Scott,James Hamilton,Bruce Given,Stacey Melquist,Rong Zhou,Ting Chang,Javier San Martin,Gerald F Watts,Ira J Goldberg,Joshua W Knowles,Robert A Hegele,Christie M Ballantyne

NEJM evidence（2023）

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摘要

APOC3-Targeting RNAi for HypertriglyceridemiaThis randomized controlled trial examined the safety and side effects of the small interfering RNA ARO-APOC3 in healthy volunteers and patients with hypertriglyceridemia and chylomicronemia. ARO-APOC3 was associated with few adverse events and no dose-limiting toxicities.

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