Towards improved shRNA and miRNA reagents as inhibitors of HIV-1 replication

被引:24
作者
Berkhout, Ben [1 ]
Liu, Ying Poi [1 ]
机构
[1] Univ Amsterdam, Acad Med Ctr, Ctr Infect & Immun Amsterdam CINIMA, Dept Med Microbiol,Lab Expt Virol, NL-1012 WX Amsterdam, Netherlands
关键词
antiviral drugs; cure for aids; drug-resistance; gene therapy; HIV1; lentiviral vector; miRNA; RNAi; shRNA; virus evolution; IMMUNODEFICIENCY-VIRUS TYPE-1; SHORT HAIRPIN RNAS; MICRORNA BIOGENESIS; LENTIVIRAL VECTORS; HEMATOPOIETIC STEM; COMBINATORIAL RNAI; CURRENT PROGRESS; GENE-THERAPY; DICER; INTERFERENCE;
D O I
10.2217/fmb.14.5
中图分类号
Q93 [微生物学];
学科分类号
071005 ; 100705 ;
摘要
miRNAs are the key players of the RNAi mechanism, which regulates the expression of a large number of mRNAs in human cells. shRNAs are man-made synthetic miRNA mimics that exploit similar intracellular RNA processing routes. Massive amounts of data derived from next-generation sequencing have revealed miRNA species that are derived from alternative biosynthesis pathways. Here, we review recent progress in our understanding of these noncanonical routes of miRNA and shRNA biosynthesis. We focus on ways to use these novel insights for the design of more potent and specific RNAi reagents for therapeutic applications, including the AgoshRNA design, which is processed differently than regular shRNAs. We will also discuss the development of a durable gene therapy against HIV1.
引用
收藏
页码:561 / 571
页数:11
相关论文
共 96 条
[1]   Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome [J].
Aiuti, Alessandro ;
Biasco, Luca ;
Scaramuzza, Samantha ;
Ferrua, Francesca ;
Cicalese, Maria Pia ;
Baricordi, Cristina ;
Dionisio, Francesca ;
Calabria, Andrea ;
Giannelli, Stefania ;
Castiello, Maria Carmina ;
Bosticardo, Marita ;
Evangelio, Costanza ;
Assanelli, Andrea ;
Casiraghi, Miriam ;
Di Nunzio, Sara ;
Callegaro, Luciano ;
Benati, Claudia ;
Rizzardi, Paolo ;
Pellin, Danilo ;
Di Serio, Clelia ;
Schmidt, Manfred ;
Von Kalle, Christof ;
Gardner, Jason ;
Mehta, Nalini ;
Neduva, Victor ;
Dow, David J. ;
Galy, Anne ;
Miniero, Roberto ;
Finocchi, Andrea ;
Metin, Ayse ;
Banerjee, Pinaki P. ;
Orange, Jordan S. ;
Galimberti, Stefania ;
Valsecchi, Maria Grazia ;
Biffi, Alessandra ;
Montini, Eugenio ;
Villa, Anna ;
Ciceri, Fabio ;
Roncarolo, Maria Grazia ;
Naldini, Luigi .
SCIENCE, 2013, 341 (6148) :865-U71
[2]   Safety and efficacy of a lentiviral vector containing three anti-HIV genes - CCR5 ribozyme, tat-rev siRNA, and TAR decoy - in SCID-hu mouse-derived T cells [J].
Anderson, Joseph ;
Li, Ming-Jie ;
Palmer, Brent ;
Remling, Leila ;
Li, Shirley ;
Yam, Priscilla ;
Yee, Jiing-Kuan ;
Rossi, John ;
Zaia, John ;
Akkina, Ramesh .
MOLECULAR THERAPY, 2007, 15 (06) :1182-1188
[3]   Beyond Secondary Structure: Primary-Sequence Determinants License Pri-miRNA Hairpins for Processing [J].
Auyeung, Vincent C. ;
Ulitsky, Igor ;
McGeary, Sean E. ;
Bartel, David P. .
CELL, 2013, 152 (04) :844-858
[4]   Evidence that HIV-1 encodes an siRNA and a suppressor of RNA silencing [J].
Bennasser, Y ;
Le, SY ;
Benkirane, M ;
Jeang, KT .
IMMUNITY, 2005, 22 (05) :607-619
[5]   MicroRNAs in viral gene regulation Preface [J].
Berkhout, B. ;
Jeang, K. -T. .
BIOCHIMICA ET BIOPHYSICA ACTA-GENE REGULATORY MECHANISMS, 2011, 1809 (11-12) :587-587
[6]   RISCy business: MicroRNAs, pathogenesis, and viruses [J].
Berkhout, Ben ;
Jeang, Kuan-Teh .
JOURNAL OF BIOLOGICAL CHEMISTRY, 2007, 282 (37) :26641-26645
[7]   Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy [J].
Biffi, Alessandra ;
Montini, Eugenio ;
Lorioli, Laura ;
Cesani, Martina ;
Fumagalli, Francesca ;
Plati, Tiziana ;
Baldoli, Cristina ;
Martino, Sabata ;
Calabria, Andrea ;
Canale, Sabrina ;
Benedicenti, Fabrizio ;
Vallanti, Giuliana ;
Biasco, Luca ;
Leo, Simone ;
Kabbara, Nabil ;
Zanetti, Gianluigi ;
Rizzo, William B. ;
Mehta, Nalini A. L. ;
Cicalese, Maria Pia ;
Casiraghi, Miriam ;
Boelens, Jaap J. ;
Del Carro, Ubaldo ;
Dow, David J. ;
Schmidt, Manfred ;
Assanelli, Andrea ;
Neduva, Victor ;
Di Serio, Clelia ;
Stupka, Elia ;
Gardner, Jason ;
von Kalle, Christof ;
Bordignon, Claudio ;
Ciceri, Fabio ;
Rovelli, Attilio ;
Roncarolo, Maria Grazia ;
Aiuti, Alessandro ;
Sessa, Maria ;
Naldini, Luigi .
SCIENCE, 2013, 341 (6148) :864-U58
[8]   A Mammalian Herpesvirus Uses Noncanonical Expression and Processing Mechanisms to Generate Viral MicroRNAs [J].
Bogerd, Hal P. ;
Karnowski, Heather W. ;
Cai, Xuezhong ;
Shin, Jinwook ;
Pohlers, Michael ;
Cullen, Bryan R. .
MOLECULAR CELL, 2010, 37 (01) :135-142
[9]   Induction of an interferon response by RNAi vectors in mammalian cells [J].
Bridge, AJ ;
Pebernard, S ;
Ducraux, A ;
Nicoulaz, AL ;
Iggo, R .
NATURE GENETICS, 2003, 34 (03) :263-264
[10]   RNA-Based Therapeutics: Current Progress and Future Prospects [J].
Burnett, John C. ;
Rossi, John J. .
CHEMISTRY & BIOLOGY, 2012, 19 (01) :60-71