Delivery technologies for genome editing

被引：414

作者：

Yin, Hao ^{[1
]}

Kauffman, Kevin J. ^{[1
,2
]}

Anderson, Daniel G. ^{[1
,2
,3
,4
]}

机构：

[1] MIT, David H Koch Inst Integrat Canc Res, Cambridge, MA 02139 USA

[2] MIT, Dept Chem Engn, Cambridge, MA 02139 USA

[3] Harvard Massachusetts Inst Technol, Div Hlth Sci & Technol, Cambridge, MA 02139 USA

[4] MIT, Inst Med Engn & Sci, Cambridge, MA 02139 USA

来源：

NATURE REVIEWS DRUG DISCOVERY | 2017年 / 16卷 / 06期

关键词：

ZINC-FINGER NUCLEASES; HEMATOPOIETIC STEM-CELLS; HEMOPHILIA GENE-THERAPY; DOUBLE-STRAND BREAKS; OFF-TARGET CLEAVAGE; HUMAN T-CELLS; IN-VIVO; MESSENGER-RNA; REPLACEMENT THERAPY; MUSCULAR-DYSTROPHY;

D O I：

10.1038/nrd.2016.280

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

With the recent development of CRISPR technology, it is becoming increasingly easy to engineer the genome. Genome-editing systems based on CRISPR, as well as transcription activator-like effector nucleases (TALENs) and zinc-finger nucleases (ZFNs), are becoming valuable tools for biomedical research, drug discovery and development, and even gene therapy. However, for each of these systems to effectively enter cells of interest and perform their function, efficient and safe delivery technologies are needed. This Review discusses the principles of biomacromolecule delivery and gene editing, examines recent advances and challenges in non-viral and viral delivery methods, and highlights the status of related clinical trials.

引用

页码：387 / 399

页数：13

共 136 条

[1] Robust ZFN-mediated genome editing in adult hemophilic mice [J].

Anguela, Xavier M. ;

Sharma, Rajiv ;

Doyon, Yannick ;

Miller, Jeffrey C. ;

Li, Hojun ;

Haurigot, Virginia ;

Rohde, Michelle E. ;

Wong, Sunnie Y. ;

Davidson, Robert J. ;

Zhou, Shangzhen ;

Gregory, Philip D. ;

Holmes, Michael C. ;

High, Katherine A. .

BLOOD, 2013, 122 (19) :3283-3287

[2] A prudent path forward for genomic engineering and germline gene modification [J].

Baltimore, David ;

Berg, Paul ;

Botchan, Michael ;

Carroll, Dana ;

Charo, R. Alta ;

Church, George ;

Corn, Jacob E. ;

Daley, George Q. ;

Doudna, Jennifer A. ;

Fenner, Marsha ;

Greely, Henry T. ;

Jinek, Martin ;

Martin, G. Steven ;

Penhoet, Edward ;

Puck, Jennifer ;

Sternberg, Samuel H. ;

Weissman, Jonathan S. ;

Yamamoto, Keith R. .

SCIENCE, 2015, 348 (6230) :36-38

[3] Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC [J].

Bankiewicz, Krystof S. ;

Forsayeth, John ;

Eberling, Jamie L. ;

Sanchez-Pernaute, Rosario ;

Pivirotto, Philip ;

Bringas, John ;

Herscovitch, Peter ;

Carson, Richard E. ;

Eckelman, William ;

Reutter, Bryan ;

Cunningham, Janet .

MOLECULAR THERAPY, 2006, 14 (04) :564-570

[4] Mutagenesis and oncogenesis by chromosomal insertion of gene transfer vectors [J].

Baum, C ;

Kustikova, O ;

Modlich, U ;

Li, ZX ;

Fehse, B .

HUMAN GENE THERAPY, 2006, 17 (03) :253-263

[5] Clinical Scale Zinc Finger Nuclease-mediated Gene Editing of PD-1 in Tumor Infiltrating Lymphocytes for the Treatment of Metastatic Melanoma [J].

Beane, Joal D. ;

Lee, Gary ;

Zheng, Zhili ;

Mendel, Matthew ;

Abate-Daga, Daniel ;

Bharathan, Mini ;

Black, Mary ;

Gandhi, Nimisha ;

Yu, Zhiya ;

Chandran, Smita ;

Giedlin, Martin ;

Ando, Dale ;

Miller, Jeff ;

Paschon, David ;

Guschin, Dmitry ;

Rebar, Edward J. ;

Reik, Andreas ;

Holmes, Michael C. ;

Gregory, Philip D. ;

Restifo, Nicholas P. ;

Rosenberg, Steven A. ;

Morgan, Richard A. ;

Feldman, Steven A. .

MOLECULAR THERAPY, 2015, 23 (08) :1380-1390

[6] Highly active zinc-finger nucleases by extended modular assembly [J].

Bhakta, Mital S. ;

Henry, Isabelle M. ;

Ousterout, David G. ;

Das, Kumitaa Theva ;

Lockwood, Sarah H. ;

Meckler, Joshua F. ;

Wallen, Mark C. ;

Zykovich, Artem ;

Yu, Yawei ;

Leo, Heather ;

Xu, Lifeng ;

Gersbach, Charles A. ;

Segal, David J. .

GENOME RESEARCH, 2013, 23 (03) :530-538

[7] DNA-binding-domain fusions enhance the targeting range and precision of Cas9 [J].

Bolukbasi, Mehmet Fatih ;

Gupta, Ankit ;

Oikemus, Sarah ;

Derr, Alan G. ;

Garber, Manuel ;

Brodsky, Michael H. ;

Zhu, Lihua Julie ;

Wolfe, Scot A. .

NATURE METHODS, 2015, 12 (12) :1150-+

[8]

Bouard D., 2000, BRIT J PHARMACOL, V157, P153

[9] Rare-disease genetics in the era of next-generation sequencing: discovery to translation [J].

Boycott, Kym M. ;

Vanstone, Megan R. ;

Bulman, Dennis E. ;

MacKenzie, Alex E. .

NATURE REVIEWS GENETICS, 2013, 14 (10) :681-691

[10] Gene Therapy for Wiskott-Aldrich Syndrome-Long-Term Efficacy and Genotoxicity [J].

Braun, Christian Joerg ;

Boztug, Kaan ;

Paruzynski, Anna ;

Witzel, Maximilian ;

Schwarzer, Adrian ;

Rothe, Michael ;

Modlich, Ute ;

Beier, Rita ;

Goehring, Gudrun ;

Steinemann, Doris ;

Fronza, Raffaele ;

Ball, Claudia Regina ;

Haemmerle, Reinhard ;

Naundorf, Sonja ;

Kuelcke, Klaus ;

Rose, Martina ;

Fraser, Chris ;

Mathias, Liesl ;

Ferrari, Rudolf ;

Abboud, Miguel R. ;

Al-Herz, Waleed ;

Kondratenko, Irina ;

Marodi, Laszlo ;

Glimm, Hanno ;

Schlegelberger, Brigitte ;

Schambach, Axel ;

Albert, Michael Heinrich ;

Schmidt, Manfred ;

von Kalle, Christof ;

Klein, Christoph .

SCIENCE TRANSLATIONAL MEDICINE, 2014, 6 (227)

← 1 2 3 4 5 6 7 8 9 10 →