CRISPR/Cas9-mediated genome editing: From basic research to translational medicine

被引：75

作者：

Jacinto, Filipe V. ^{[1
,2
,3
]}

Link, Wolfgang ^{[4
]}

Ferreira, Bibiana I. ^{[1
,2
,3
]}

机构：

[1] Ctr Biomed Res CBMR, Bldg 2,Gambelas Campus, P-8005139 Faro, Portugal

[2] Univ Algarve, Dept Med & Ciencias Biomed DCBM, Faro, Portugal

[3] ABC, Faro, Portugal

[4] UAM, CSIC, Inst Invest Biomed Alberto Sols, Arturo Duperier 4, Madrid 28029, Spain

来源：

JOURNAL OF CELLULAR AND MOLECULAR MEDICINE | 2020年 / 24卷 / 07期

关键词：

CRISPR; gene therapy; genome editing; translational medicine; PLURIPOTENT STEM-CELL; STRAND BREAK REPAIR; HOMOLOGY-DIRECTED REPAIR; ZINC-FINGER NUCLEASES; CRISPR-CAS; T-CELLS; BINDING-SPECIFICITY; GENE ACTIVATION; DNA-SEQUENCES; WIDE ANALYSIS;

D O I：

10.1111/jcmm.14916

中图分类号：

Q2 [细胞生物学];

学科分类号：

071009 ; 090102 ;

摘要：

The recent development of the CRISPR/Cas9 system as an efficient and accessible programmable genome-editing tool has revolutionized basic science research. CRISPR/Cas9 system-based technologies have armed researchers with new powerful tools to unveil the impact of genetics on disease development by enabling the creation of precise cellular and animal models of human diseases. The therapeutic potential of these technologies is tremendous, particularly in gene therapy, in which a patient-specific mutation is genetically corrected in order to treat human diseases that are untreatable with conventional therapies. However, the translation of CRISPR/Cas9 into the clinics will be challenging, since we still need to improve the efficiency, specificity and delivery of this technology. In this review, we focus on several in vitro, in vivo and ex vivo applications of the CRISPR/Cas9 system in human disease-focused research, explore the potential of this technology in translational medicine and discuss some of the major challenges for its future use in patients.

引用

页码：3766 / 3778

页数：13

共 169 条

[1] RNA targeting with CRISPR-Cas13 [J].

Abudayyeh, Omar O. ;

Gootenberg, Jonathan S. ;

Essletzbichler, Patrick ;

Han, Shuo ;

Joung, Julia ;

Belanto, Joseph J. ;

Verdine, Vanessa ;

Cox, David B. T. ;

Kellner, Max J. ;

Regev, Aviv ;

Lander, Eric S. ;

Voytas, Daniel F. ;

Ting, Alice Y. ;

Zhang, Feng .

NATURE, 2017, 550 (7675) :280-+

[2] C2c2 is a single-component programmable RNA-guided RNA-targeting CRISPR effector [J].

Abudayyeh, Omar O. ;

Gootenberg, Jonathan S. ;

Konermann, Silvana ;

Joung, Julia ;

Slaymaker, Ian M. ;

Cox, David B. T. ;

Shmakov, Sergey ;

Makarova, Kira S. ;

Semenova, Ekaterina ;

Minakhin, Leonid ;

Severinov, Konstantin ;

Regev, Aviv ;

Lander, Eric S. ;

Koonin, Eugene V. ;

Zhang, Feng .

SCIENCE, 2016, 353 (6299)

[3] CRISPR provides acquired resistance against viruses in prokaryotes [J].

Barrangou, Rodolphe ;

Fremaux, Christophe ;

Deveau, Helene ;

Richards, Melissa ;

Boyaval, Patrick ;

Moineau, Sylvain ;

Romero, Dennis A. ;

Horvath, Philippe .

SCIENCE, 2007, 315 (5819) :1709-1712

[4] A decade of discovery: CRISPR functions and applications [J].

Barrangou, Rodolphe ;

Horvath, Philippe .

NATURE MICROBIOLOGY, 2017, 2 (07)

[5] Enhancing gene targeting with designed zinc finger nucleases [J].

Bibikova, M ;

Beumer, K ;

Trautman, JK ;

Carroll, D .

SCIENCE, 2003, 300 (5620) :764-764

[6] Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors [J].

Boch, Jens ;

Scholze, Heidi ;

Schornack, Sebastian ;

Landgraf, Angelika ;

Hahn, Simone ;

Kay, Sabine ;

Lahaye, Thomas ;

Nickstadt, Anja ;

Bonas, Ulla .

SCIENCE, 2009, 326 (5959) :1509-1512

[7] Clustered regularly interspaced short palindrome repeats (CRISPRs) have spacers of extrachromosomal origin [J].

Bolotin, A ;

Ouinquis, B ;

Sorokin, A ;

Ehrlich, SD .

MICROBIOLOGY-SGM, 2005, 151 :2551-2561

[8] Small CRISPR RNAs guide antiviral defense in prokaryotes [J].

Brouns, Stan J. J. ;

Jore, Matthijs M. ;

Lundgren, Magnus ;

Westra, Edze R. ;

Slijkhuis, Rik J. H. ;

Snijders, Ambrosius P. L. ;

Dickman, Mark J. ;

Makarova, Kira S. ;

Koonin, Eugene V. ;

van der Oost, John .

SCIENCE, 2008, 321 (5891) :960-964

[9] Gene targeting in mice: functional analysis of the mammalian genome for the twenty-first century [J].

Capecchi, MR .

NATURE REVIEWS GENETICS, 2005, 6 (06) :507-512

[10] ALTERING THE GENOME BY HOMOLOGOUS RECOMBINATION [J].

CAPECCHI, MR .

SCIENCE, 1989, 244 (4910) :1288-1292

← 1 2 3 4 5 6 7 8 9 10 →