Therapeutic applications of noncoding RNAs

被引:18
作者
Latronico, Michael V. G. [1 ]
Condorelli, Gianluigi [1 ,2 ,3 ]
机构
[1] Humanitas Res Hosp, Milan, Italy
[2] Univ Milan, Milan, Italy
[3] Natl Res Council Italy, Milan, Italy
基金
欧洲研究理事会;
关键词
heart; long noncoding RNAs; microRNAs; CARDIOVASCULAR-DISEASE; CARDIAC-HYPERTROPHY; SKELETAL-MUSCLE; MICRORNA; INHIBITION; EXPRESSION; DELIVERY; LIVER; DESIGN;
D O I
10.1097/HCO.0000000000000162
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Purpose of review In this review, we summarize the basic principles underlying the therapeutic use of nonprotein coding (nc) RNAs, such as microRNA (miRNA) and long noncoding RNA, in the cardiovascular field, focusing, where possible, on recent advances that may lead to translation to the clinic for heart failure. Recent findings The number of individual miRNAs associated with a given aspect of heart disease is increasing rapidly, as is the data on miRNA profiles in normal and diseased myocardium. Less is known on the role of long noncoding RNA, and to date only a few have been studied in the heart. Novel oligonucleotide-based therapies have started to trickle into the clinic, but strategies focusing on ncRNA are still in a clinical/preclinical trial phase. Summary The discovery of functional ncRNAs is leading to a better understanding of the mechanisms underlying cardiovascular physiology. Dysregulation of ncRNAs is being increasingly associated with many diseases affecting the heart and in certain instances may have a pathogenic role. Therapeutic intervention aimed at opposing ncRNA misexpression has been widely demonstrated to be effective in blunting disease in animal models, and may thus have potential in the clinical setting.
引用
收藏
页码:213 / 221
页数:9
相关论文
共 56 条
[1]   An Emerging Adeno-Associated Viral Vector Pipeline for Cardiac Gene Therapy [J].
Asokan, Aravind ;
Samulski, R. Jude .
HUMAN GENE THERAPY, 2013, 24 (11) :906-913
[2]   Therapeutic inhibition of the miR-34 family attenuates pathological cardiac remodeling and improves heart function [J].
Bernardo, Bianca C. ;
Gao, Xiao-Ming ;
Winbanks, Catherine E. ;
Boey, Esther J. H. ;
Tham, Yow Keat ;
Kiriazis, Helen ;
Gregorevic, Paul ;
Obad, Susanna ;
Kauppinen, Sakari ;
Du, Xiao-Jun ;
Lin, Ruby C. Y. ;
McMullen, Julie R. .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2012, 109 (43) :17615-17620
[3]   Cardiac Gene Transfer of Short Hairpin RNA Directed Against Phospholamban Effectively Knocks Down Gene Expression but Causes Cellular Toxicity in Canines [J].
Bish, Lawrence T. ;
Sleeper, Meg M. ;
Reynolds, Caryn ;
Gazzara, Jeffrey ;
Withnall, Elanor ;
Singletary, Gretchen E. ;
Buchlis, George ;
Hui, Daniel ;
High, Katherine A. ;
Gao, Guangping ;
Wilson, James M. ;
Sweeney, H. Lee .
HUMAN GENE THERAPY, 2011, 22 (08) :969-977
[4]   First microRNA mimic enters clinic [J].
Bouchie, Aaron .
NATURE BIOTECHNOLOGY, 2013, 31 (07) :577-577
[5]   Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer [J].
Buchlis, George ;
Podsakoff, Gregory M. ;
Radu, Antonetta ;
Hawk, Sarah M. ;
Flake, Alan W. ;
Mingozzi, Federico ;
High, Katherine A. .
BLOOD, 2012, 119 (13) :3038-3041
[6]   MicroRNA-133 controls cardiac hypertrophy [J].
Care, Alessandra ;
Catalucci, Daniele ;
Felicetti, Federica ;
Bonci, Desiree ;
Addario, Antonio ;
Gallo, Paolo ;
Bang, Marie-Louise ;
Segnalini, Patrizia ;
Gu, Yusu ;
Dalton, Nancy D. ;
Elia, Leonardo ;
Latronico, Michael V. G. ;
Hoydal, Morten ;
Autore, Camillo ;
Russo, Matteo A. ;
Dorn, Gerald W., II ;
Ellingsen, Oyvind ;
Ruiz-Lozano, Pilar ;
Peterson, Kirk L. ;
Croce, Carlo M. ;
Peschle, Cesare ;
Condorelli, Gianluigi .
NATURE MEDICINE, 2007, 13 (05) :613-618
[7]   Strand-specific 5′-O-methylation of siRNA duplexes controls guide strand selection and targeting specificity [J].
Chen, Po Yu ;
Weinmann, Lasse ;
Gaidatzis, Dimos ;
Pei, Yi ;
Zavolan, Mihaela ;
Tuschl, Thomas ;
Meister, Gunter .
RNA, 2008, 14 (02) :263-274
[8]   siRNA function in RNAi: A chemical modification analysis [J].
Chiu, YL ;
Rana, TM .
RNA, 2003, 9 (09) :1034-1048
[9]   MicroRNAs and their isomiRs function cooperatively to target common biological pathways [J].
Cloonan, Nicole ;
Wani, Shivangi ;
Xu, Qinying ;
Gu, Jian ;
Lea, Kristi ;
Heater, Sheila ;
Barbacioru, Catalin ;
Steptoe, Anita L. ;
Martin, Hilary C. ;
Nourbakhsh, Ehsan ;
Krishnan, Keerthana ;
Gardiner, Brooke ;
Wang, Xiaohui ;
Nones, Katia ;
Steen, Jason A. ;
Matigian, Nicholas A. ;
Wood, David L. ;
Kassahn, Karin S. ;
Waddell, Nic ;
Shepherd, Jill ;
Lee, Clarence ;
Ichikawa, Jeff ;
McKernan, Kevin ;
Bramlett, Kelli ;
Kuersten, Scott ;
Grimmond, Sean M. .
GENOME BIOLOGY, 2011, 12 (12)
[10]   microRNAs in Cardiovascular Diseases Current Knowledge and the Road Ahead [J].
Condorelli, Gianluigi ;
Latronico, Michael V. G. ;
Cavarretta, Elena .
JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY, 2014, 63 (21) :2177-2187