CRISPR/Cas9 technology as a potent molecular tool for gene therapy

被引:102
作者
Karimian, Ansar [1 ,2 ,3 ]
Azizian, Khalil [4 ]
Parsian, Hadi [1 ]
Rafieian, Sona [5 ]
Shafiei-Irannejad, Vahid [6 ]
Kheyrollah, Maryam [7 ]
Yousefi, Mehdi [8 ,9 ]
Majidinia, Maryam [10 ]
Yousefi, Bahman [11 ,12 ]
机构
[1] Babol Univ Med Sci, Hlth Res Inst, Cellular & Mol Biol Res Ctr, Babol Sar, Iran
[2] Kurdistan Univ Med Sci, Canc & Immunol Res Ctr, Sanandaj, Iran
[3] Babol Univ Med Sci, Student Res Comm, Babol Sar, Iran
[4] Tabriz Univ Med Sci, Liver & Gastrointestinal Dis Res Ctr, Tabriz, Iran
[5] Zanjan Univ Med Sci, Sch Dent, Dept Oral & Maxillofacial Pathol, Zanjan, Iran
[6] Urmia Univ Med Sci, Cellular & Mol Res Ctr, Orumiyeh, Iran
[7] Natl Inst Genet Engn & Biotechnol, Dept Mol Med, Tehran, Iran
[8] Tabriz Univ Med Sci, Mol Med Res Ctr, Tabriz, Iran
[9] Tabriz Univ Med Sci, Immunol Res Ctr, Tabriz, Iran
[10] Urmia Univ Med Sci, Tumor Res Ctr, Orumiyeh, Iran
[11] Baqiyatallah Univ Med Sci, Appl Biotechnol Res Ctr, Tehran, Iran
[12] Tabriz Univ Med Sci, Fac Med, Dept Biochem & Clin Labs, Tabriz 5166614766, Iran
关键词
cancer treatment; CRISPR-Cas9; gene therapy; genome editing; GENOME EDITING TECHNOLOGIES; IN-VIVO; SIGNALING PATHWAY; VIRAL VECTORS; CRISPR-CAS9; CELLS; DELIVERY; RESISTANCE; SYSTEM; CLASSIFICATION;
D O I
10.1002/jcp.27972
中图分类号
Q2 [细胞生物学];
学科分类号
071009 ; 090102 ;
摘要
Clustered regularly interspaced short palindromic repeats/CRISPR-associated nuclease 9 (CRISPR-Cas9) is an RNA-guided gene editing tool which offers several advantageous characteristics in comparison with the conventional methods (e.g., zinc finger nucleases and transcription activator-like effector nucleases) such as cost-effectiveness, flexibility, and being easy-to-use. Despite some limitations such as efficient delivery and safety, CRISPR-Cas9 is still the most convenient tool for gene editing purposes. Due to the potential capability of the CRISPR-Cas9 system in genome editing and correction of casual mutations, it can be considered as a possible therapeutic system in the treatment of disorders associated with the genome mutations and in particular cancer treatment. In this review, we will discuss CRISPR-Cas-based gene editing along with its classifications and mechanism of action. Furthermore, the therapeutic application of the CRISPR-Cas9 system in mutational disorders, delivery systems, as well as its advantages and limitations with a special emphasis on cancer treatment will be discussed.
引用
收藏
页码:12267 / 12277
页数:11
相关论文
共 87 条
[1]   Clustered regularly interspaced short palindromic repeats (CRISPRs): the hallmark of an ingenious antiviral defense mechanism in prokaryotes [J].
Al-Attar, Sinan ;
Westra, Edze R. ;
van der Oost, John ;
Brouns, Stan J. J. .
BIOLOGICAL CHEMISTRY, 2011, 392 (04) :277-289
[2]   CRISPR-Cas adaptation: insights into the mechanism of action [J].
Amitai, Gil ;
Sorek, Rotem .
NATURE REVIEWS MICROBIOLOGY, 2016, 14 (02) :67-76
[3]   Suppression of p53R2 gene expression with specific siRNA sensitizes HepG2 cells to doxorubicin [J].
Azimi, Ako ;
Majidinia, Maryam ;
Shafiei-Irannejad, Vahid ;
Jahanban-Esfahlan, Rana ;
Ahmadi, Yasin ;
Karimian, Ansar ;
Mir, Seyed Mostafa ;
Karami, Hadi ;
Yousefi, Bahman .
GENE, 2018, 642 :249-255
[4]   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
[5]   Structure and activity of the Cas3 HD nuclease MJ0384, an effector enzyme of the CRISPR interference [J].
Beloglazova, Natalia ;
Petit, Pierre ;
Flick, Robert ;
Brown, Greg ;
Savchenko, Alexei ;
Yakunin, Alexander F. .
EMBO JOURNAL, 2011, 30 (22) :4616-4627
[6]   Classification and evolution of type II CRISPR-Cas systems [J].
Chylinski, Krzysztof ;
Makarova, Kira S. ;
Charpentier, Emmanuelle ;
Koonin, Eugene V. .
NUCLEIC ACIDS RESEARCH, 2014, 42 (10) :6091-6105
[7]   Multiplex Genome Engineering Using CRISPR/Cas Systems [J].
Cong, Le ;
Ran, F. Ann ;
Cox, David ;
Lin, Shuailiang ;
Barretto, Robert ;
Habib, Naomi ;
Hsu, Patrick D. ;
Wu, Xuebing ;
Jiang, Wenyan ;
Marraffini, Luciano A. ;
Zhang, Feng .
SCIENCE, 2013, 339 (6121) :819-823
[8]   Exosomes: natural nanoparticles as bio shuttles for RNAi delivery [J].
Darband, Saber Ghazizadeh ;
Mirza-Aghazadeh-Attari, Mohammad ;
Kaviani, Mojtaba ;
Mihanfar, Ainaz ;
Sadighparvar, Shirin ;
Yousefi, Bahman ;
Majidinia, Maryam .
JOURNAL OF CONTROLLED RELEASE, 2018, 289 :158-170
[9]   Current prospects for RNA interference-based therapies [J].
Davidson, Beverly L. ;
McCray, Paul B., Jr. .
NATURE REVIEWS GENETICS, 2011, 12 (05) :329-340
[10]   Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing [J].
Ding, Qiurong ;
Strong, Alanna ;
Patel, Kevin M. ;
Ng, Sze-Ling ;
Gosis, Bridget S. ;
Regan, Stephanie N. ;
Cowan, Chad A. ;
Rader, Daniel J. ;
Musunuru, Kiran .
CIRCULATION RESEARCH, 2014, 115 (05) :488-+