Clinical advances of siRNA therapeutics

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作者信息关键词期刊信息基础信息归属信息摘要

成果类型：

期刊论文

作者：

Hu, Bo;Weng, Yuhua;Xia, Xin-Hua;Liang, Xing-jie;Huang, Yuanyu

通讯作者：

Huang, YY

作者机构：

[Weng, Yuhua; Huang, YY; Huang, Yuanyu; Hu, Bo] Beijing Inst Technol, Sch Life Sci, Adv Res Inst Multidisciplinary Sci, Key Lab Mol Med & Biotherapy, 5 Zhongguancun South St, Beijing 100081, Peoples R China.

[Huang, Yuanyu; Xia, Xin-Hua] Hunan Univ Chinese Med, Sch Pharm, Changsha 410208, Hunan, Peoples R China.

[Liang, Xing-jie] Chinese Acad Sci, CAS Ctr Excellence Nanosci, Natl Ctr Nanosci & Technol China, Key Lab Biomed Effects Nanomat & Nanosafety, Beijing 100190, Peoples R China.

通讯机构：

[Huang, YY ] B

Beijing Inst Technol, Sch Life Sci, Adv Res Inst Multidisciplinary Sci, Key Lab Mol Med & Biotherapy, 5 Zhongguancun South St, Beijing 100081, Peoples R China.

语种：

英文

关键词：

aln aat 02;aln as 1;aln cc 5;aln g 01;aln ttr 02;aln ttrsc 01;amg 890;antisense oligonucleotide;arc 520;arc aat;aro ang 3;aro apoc 3;asvasiran;cemdisiran;cholesterol;dcr ph 1;double stranded RNA;fitusiran;givosiran;inclisiran;lipid;liposome;lumasiran;n acetylgalactosamine;patisiran;pf 04523655;pro 040201;qpi 1007;revusiran;small interfering RNA;solid lipid nanoparticle;syl 1001;TKM Ebola;unclassified drug;unindexed drug;vutrisiran;galactose;ligand;patisiran;small interfering RNA;acute intermittent porphyria;alpha 1 antitrypsin deficiency;amyloidosis;anterior ischemic optic neuropathy;ATTR amyloidosis;cardiovascular disease;diabetic macular edema;diabetic retinopathy;drug clearance;drug half life;drug targeting;dry eye;Ebola hemorrhagic fever;gene activity;gene expression;gene silencing;hemolytic uremic syndrome;hemophilia;hepatitis B;human;hypercholesterolemia;hypertriglyceridemia;liver disease;nanoencapsulation;nonhuman;oxalosis 1;priority journal;respiratory syncytial virus infection;Review;RNAi therapeutics;subretinal neovascularization;translation regulation;chemistry;gene transfer;genetics;metabolism;RNA interference;Galactose;Gene Transfer Techniques;Humans;Ligands;Liposomes;RNA Interference;RNA, Small Interfering

期刊：

JOURNAL OF GENE MEDICINE

ISSN：

1099-498X

年：

2019

卷：

期：

页码：

e3097-

DOI：

10.1002/jgm.3097

基金类别：

This work was supported by the National Natural Science Foundation of China (31871003), the Beijing Institute of Technology Research Fund Program for Young Scholars and the Fundamental Research Funds for the Central Universities, Hunan Provincial Natural Science Foundation of China (2018JJ1019, 2019JJ50196), the Hu‐Xiang Young Talent Program (2018RS3094), and the Postdoctoral Science Foundation of China (2018 M630085). BH, YW and YH wrote the paper. XHX and XJL provided insightful discussions and suggestions. YH led the project. This work was supported by the National Natural Science Foundation of China (31871003), the Beijing Institute of Technology Research Fund Program for Young Scholars and the Fundamental Research Funds for the Central Universities, Hunan Provincial Natural Science Foundation of China (2018JJ1019, 2019JJ50196), the Hu-Xiang Young Talent Program (2018RS3094), and the Postdoctoral Science Foundation of China (2018?M630085). BH, YW and YH wrote the paper. XHX and XJL provided insightful discussions and suggestions. YH led the project.

机构署名：

本校为其他机构

院系归属：

药学院

摘要：

Small interfering RNA (siRNA) enables efficient target gene silencing by employing a RNA interference (RNAi) mechanism, which can compromise gene expression and regulate gene activity by cleaving mRNA or repressing its translation. Twenty years after the discovery of RNAi in 1998, ONPATTRO™ (patisiran) (Alnylam Pharmaceuticals, Inc.), a lipid formulated siRNA modality, was approved for the first time by United States Food and Drug Administration and the European Commission in 2018. With this milestone achievement, siRNA therapeutics will soar ...

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