[PDF][PDF] 首例RNA 干扰药物问世及该领域技术演化历程
黄渊余 - Progress in Biochemistry and Biophysics, 2019 - researchgate.net
摘要近期, RNA 干扰(RNAi) 制药公司Alnylam 开发的小干扰RNA (siRNA) 药物ONPATTRO™(
Patisiran) 相继获得美国食品与药物管理局和欧盟委员会批准上市, 用于治疗成人患者的遗传性 …
Patisiran) 相继获得美国食品与药物管理局和欧盟委员会批准上市, 用于治疗成人患者的遗传性 …
RNAi: a potential new class of therapeutic for human genetic disease
AA Seyhan - Human genetics, 2011 - Springer
Dominant negative genetic disorders, in which a mutant allele of a gene causes disease in
the presence of a second, normal copy, have been challenging since there is no cure and …
the presence of a second, normal copy, have been challenging since there is no cure and …
RNAi therapeutic and its innovative biotechnological evolution
Abstract Recently, United States Food and Drug Administration (FDA) and European
Commission (EC) approved Alnylam Pharmaceuticals' RNA interference (RNAi) therapeutic …
Commission (EC) approved Alnylam Pharmaceuticals' RNA interference (RNAi) therapeutic …
RNAi hits another rut
A Mullard - Nature Reviews Drug Discovery, 2016 - go.gale.com
RNA interference (RNAi) company Alnylam Pharmaceuticals has discontinued one of its
lead programmes, halting the development of phase III candidate revusiran due to an" …
lead programmes, halting the development of phase III candidate revusiran due to an" …
Update on the clinical utility of an RNA interference-based treatment: focus on Patisiran
M Rizk, Ş Tüzmen - Pharmacogenomics and personalized …, 2017 - Taylor & Francis
RNA interference (RNAi) is a naturally existing endogenous mechanism for post-
transcriptional gene regulation, nowadays commonly utilized for functional characterization …
transcriptional gene regulation, nowadays commonly utilized for functional characterization …