Enabling functional genomics with genome engineering

被引:80
作者
Hilton, Isaac B. [1 ,2 ]
Gersbach, Charles A. [1 ,2 ,3 ]
机构
[1] Duke Univ, Dept Biomed Engn, Durham, NC 27708 USA
[2] Duke Univ, Ctr Genom & Computat Biol, Durham, NC 27708 USA
[3] Duke Univ, Med Ctr, Dept Orthopaed Surg, Durham, NC 27710 USA
基金
美国国家科学基金会; 美国国家卫生研究院;
关键词
ZINC-FINGER NUCLEASES; DNA-BINDING SPECIFICITY; PLURIPOTENT STEM-CELLS; DOUBLE-STRAND BREAKS; OFF-TARGET CLEAVAGE; GENE-EXPRESSION; TRANSCRIPTION FACTORS; CRYSTAL-STRUCTURE; WIDE ANALYSIS; GUIDE RNA;
D O I
10.1101/gr.190124.115
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
Advances in genome engineering technologies have made the precise control over genome sequence and regulation possible across a variety of disciplines. These tools can expand our understanding of fundamental biological processes and create new opportunities for therapeutic designs. The rapid evolution of these methods has also catalyzed a new era of genomics that includes multiple approaches to functionally characterize and manipulate the regulation of genomic information. Here, we review the recent advances of the most widely adopted genome engineering platforms and their application to functional genomics. This includes engineered zinc finger proteins, TALEs/TALENs, and the CRISPR / Cas9 system as nucleases for genome editing, transcription factors for epigenome editing, and other emerging applications. We also present current and potential future applications of these tools, as well as their current limitations and areas for future advances.
引用
收藏
页码:1442 / 1455
页数:14
相关论文
共 274 条
[41]   DNA-binding specificity is a major determinant of the activity and toxicity of zinc-finger Nucleases [J].
Cornu, Tatjana I. ;
Thibodeau-Beganny, Stacey ;
Guhl, Eva ;
Alwin, Stephen ;
Eichtinger, Magdalena ;
Joung, J. K. ;
Cathomen, Toni .
MOLECULAR THERAPY, 2008, 16 (02) :352-358
[42]   Thymine DNA Glycosylase Is Essential for Active DNA Demethylation by Linked Deamination-Base Excision Repair [J].
Cortellino, Salvatore ;
Xu, Jinfei ;
Sannai, Mara ;
Moore, Robert ;
Caretti, Elena ;
Cigliano, Antonio ;
Le Coz, Madeleine ;
Devarajan, Karthik ;
Wessels, Andy ;
Soprano, Dianne ;
Abramowitz, Lara K. ;
Bartolomei, Marisa S. ;
Rambow, Florian ;
Bassi, Maria Rosaria ;
Bruno, Tiziana ;
Fanciulli, Maurizio ;
Renner, Catherine ;
Klein-Szanto, Andres J. ;
Matsumoto, Yoshihiro ;
Kobi, Dominique ;
Davidson, Irwin ;
Alberti, Christophe ;
Larue, Lionel ;
Bellacosa, Alfonso .
CELL, 2011, 146 (01) :67-79
[43]   Therapeutic genome editing: prospects and challenges [J].
Cox, David Benjamin Turitz ;
Platt, Randall Jeffrey ;
Zhang, Feng .
NATURE MEDICINE, 2015, 21 (02) :121-131
[44]   COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites [J].
Cradick, Thomas J. ;
Qiu, Peng ;
Lee, Ciaran M. ;
Fine, Eli J. ;
Bao, Gang .
MOLECULAR THERAPY-NUCLEIC ACIDS, 2014, 3 :e214
[45]   Targeted Correction and Restored Function of the CFTR Gene in Cystic Fibrosis Induced Pluripotent Stem Cells [J].
Crane, Ana M. ;
Kramer, Philipp ;
Bui, Jacquelin H. ;
Chung, Wook Joon ;
Li, Xuan Shirley ;
Gonzalez-Garay, Manuel L. ;
Hawkins, Finn ;
Liao, Wei ;
Mora, Daniela ;
Choi, Sangbum ;
Wang, Jianbin ;
Sun, Helena C. ;
Paschon, David E. ;
Guschin, Dmitry Y. ;
Gregory, Philip D. ;
Kotton, Darrell N. ;
Holmes, Michael C. ;
Sorscher, Eric J. ;
Davis, Brian R. .
STEM CELL REPORTS, 2015, 4 (04) :569-577
[46]   Condensin-driven remodelling of X chromosome topology during dosage compensation [J].
Crane, Emily ;
Bian, Qian ;
McCord, Rachel Patton ;
Lajoie, Bryan R. ;
Wheeler, Bayly S. ;
Ralston, Edward J. ;
Uzawa, Satoru ;
Dekker, Job ;
Meyer, Barbara J. .
NATURE, 2015, 523 (7559) :240-U299
[47]   Targeted integration in rat and mouse embryos with zinc- finger nucleases [J].
Cui, Xiaoxia ;
Ji, Diana ;
Fisher, Daniel A. ;
Wu, Yumei ;
Briner, David M. ;
Weinstein, Edward J. .
NATURE BIOTECHNOLOGY, 2011, 29 (01) :64-+
[48]   Super-muscly pigs created by small genetic tweak [J].
Cyranoski, David .
NATURE, 2015, 523 (7558) :13-14
[49]   Modular Extracellular Sensor Architecture for Engineering Mammalian Cell-based Devices [J].
Daringer, Nichole M. ;
Dudek, Rachel M. ;
Schwarz, Kelly A. ;
Leonard, Joshua N. .
ACS SYNTHETIC BIOLOGY, 2014, 3 (12) :892-902
[50]  
Davis KM, 2015, NAT CHEM BIOL, V11, P316, DOI [10.1038/NCHEMBIO.1793, 10.1038/nchembio.1793]