Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria
卟啉
急性间歇性卟啉症
RNA干扰
医学
核糖核酸
基因
内科学
生物
遗传学
作者
Eliane Sardh,Pauline Harper,Manisha Balwani,Penelope E. Stein,David C. Rees,D. Montgomery Bissell,Robert J. Desnick,Charles Parker,John D. Phillips,Herbert L. Bonkovsky,Daphne Vassiliou,Craig Penz,Amy Chan-Daniels,Qiuling He,William Querbes,Kevin Fitzgerald,Jae Bum Kim,Pushkal Garg,Akshay Vaishnaw,Amy Simon
Induction of delta aminolevulinic acid synthase 1 (ALAS1) gene expression and accumulation of neurotoxic intermediates result in neurovisceral attacks and disease manifestations in patients with acute intermittent porphyria, a rare inherited disease of heme biosynthesis. Givosiran is an investigational RNA interference therapeutic agent that inhibits hepatic ALAS1 synthesis.
SYLH
科研小民工
杳鸢
劲秉
迟大猫
Zn
小林太郎
故意的傲玉
sweetbearm
lalala
36456657
RC_Wang
浦肯野
kingwill
iNk
火星上的菲鹰
pluto
从心
坚强的广山
maox1aoxin
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