In vivo genome editing via the HITI method as a tool for gene therapy

被引：89

作者：

Suzuki, Keiichiro ^{[1
,2
,3
]}

Belmonte, Juan Carlos Izpisua ^{[3
]}

机构：

[1] Osaka Univ, Inst Adv Cocreat Studies, Suita, Osaka, Japan

[2] Osaka Univ, Grad Sch Engn Sci, Toyonaka, Osaka, Japan

[3] Salk Inst Biol Studies, Gene Express Lab, 10010 N Torrey Pines Rd, La Jolla, CA 92037 USA

来源：

JOURNAL OF HUMAN GENETICS | 2018年 / 63卷 / 02期

关键词：

LEBERS CONGENITAL AMAUROSIS; MEDIATED TARGETED INTEGRATION; KNOCK-IN; CRISPR/CAS9; SYSTEM; MOUSE MODEL; DNA-REPAIR; ZEBRAFISH; CELLS; RECOMBINATION; CRISPR-CAS9;

D O I：

10.1038/s10038-017-0352-4

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

Using genome-editing technologies to correct specific mutations represents a potentially transformative new approach for treating genetic disorders. Despite rapid advances in the field of genome editing, it is still unclear whether the long-standing goal of in vivo targeted transgene integration is feasible. This is primarily because current tools are inefficient. In particular, current technologies are incapable of targeted gene knock-in in non-dividing cells, the major building blocks of adult tissues. This poses a significant barrier for developing therapeutic strategies to treat a broad range of devastating genetic disorders. Recently, our group has developed a unique CRISPR/Cas9-based strategy, termed homology-independent targeted insertion (HITI), which enables targeted gene insertion in non-dividing cells, both in vitro and in vivo. This review will summarize current progress in developing this technology, and discuss the potential impact of HITI-based gene-correction therapies.

引用

页码：157 / 164

页数：8

共 51 条

[1] Highly efficient CRISPR/Cas9-mediated knock-in in zebrafish by homology-independent DNA repair [J].

Auer, Thomas O. ;

Duroure, Karine ;

De Cian, Anne ;

Concordet, Jean-Paul ;

Del Bene, Filippo .

GENOME RESEARCH, 2014, 24 (01) :142-153

[2] Long-Term Effect of Gene Therapy on Leber's Congenital Amaurosis [J].

Bainbridge, J. W. B. ;

Mehat, M. S. ;

Sundaram, V. ;

Robbie, S. J. ;

Barker, S. E. ;

Ripamonti, C. ;

Georgiadis, A. ;

Mowat, F. M. ;

Beattie, S. G. ;

Gardner, P. J. ;

Feathers, K. L. ;

Luong, V. A. ;

Yzer, S. ;

Balaggan, K. ;

Viswanathan, A. ;

de Ravel, T. J. L. ;

Casteels, I. ;

Holder, G. E. ;

Tyler, N. ;

Fitzke, F. W. ;

Weleber, R. G. ;

Nardini, M. ;

Moore, A. T. ;

Thompson, D. A. ;

Petersen-Jones, S. M. ;

Michaelides, M. ;

van den Born, L. I. ;

Stockman, A. ;

Smith, A. J. ;

Rubin, G. ;

Ali, R. R. .

NEW ENGLAND JOURNAL OF MEDICINE, 2015, 372 (20) :1887-1897

[3] Effect of gene therapy on visual function in Leber's congenital amaurosis [J].

Bainbridge, James W. B. ;

Smith, Alexander J. ;

Barker, Susie S. ;

Robbie, Scott ;

Henderson, Robert ;

Balaggan, Kamaljit ;

Viswanathan, Ananth ;

Holder, Graham E. ;

Stockman, Andrew ;

Tyler, Nick ;

Petersen-Jones, Simon ;

Bhattacharya, Shomi S. ;

Thrasher, Adrian J. ;

Fitzke, Fred W. ;

Carter, Barrie J. ;

Rubin, Gary S. ;

Moore, Anthony T. ;

Ali, Robin R. .

NEW ENGLAND JOURNAL OF MEDICINE, 2008, 358 (21) :2231-2239

[4] In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa [J].

Bakondi, Benjamin ;

Lv, Wenjian ;

Lui, Bin ;

Jones, Melissa K. ;

Tsai, Yuchun ;

Kim, Kevin J. ;

Levy, Rachelle ;

Akhtar, Aslam Abbasi ;

Breunig, Joshua J. ;

Svendseni, Clive N. ;

Wang, Shaomei .

MOLECULAR THERAPY, 2016, 24 (03) :556-563

[5] Multiplexed Targeted Genome Engineering Using a Universal Nuclease-Assisted Vector Integration System [J].

Brown, Alexander ;

Woods, Wendy S. ;

Perez-Pinera, Pablo .

ACS SYNTHETIC BIOLOGY, 2016, 5 (07) :582-588

[6] 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

[7] Therapeutic genome editing: prospects and challenges [J].

Cox, David Benjamin Turitz ;

Platt, Randall Jeffrey ;

Zhang, Feng .

NATURE MEDICINE, 2015, 21 (02) :121-131

[8] In vivo cleavage of transgene donors promotes nuclease-mediated targeted integration [J].

Cristea, Sandra ;

Freyvert, Yevgeniy ;

Santiago, Yolanda ;

Holmes, Michael C. ;

Urnov, Fyodor D. ;

Gregory, Philip D. ;

Cost, Gregory J. .

BIOTECHNOLOGY AND BIOENGINEERING, 2013, 110 (03) :871-880

[9] Mutation of the receptor tyrosine kinase gene Mertk in the retinal dystrophic RCS rat [J].

D'Cruz, PM ;

Yasumura, D ;

Weir, J ;

Matthes, MT ;

Abderrahim, H ;

LaVail, MM ;

Vollrath, D .

HUMAN MOLECULAR GENETICS, 2000, 9 (04) :645-651

[10] In vivo blunt-end cloning through CRISPR/Cas9-facilitated non-homologous end-joining [J].

Geisinger, Jonathan M. ;

Turan, Soren ;

Hernandez, Sophia ;

Spector, Laura P. ;

Calos, Michele P. .

NUCLEIC ACIDS RESEARCH, 2016, 44 (08)

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