Delivery and Specificity of CRISPR/Cas9 Genome Editing Technologies for Human Gene Therapy

被引：152

作者：

Gori, Jennifer L. ^{[1
]}

Hsu, Patrick D. ^{[1
]}

Maeder, Morgan L. ^{[1
]}

Shen, Shen ^{[1
]}

Welstead, G. Grant ^{[1
]}

Bumcrot, David ^{[1
]}

机构：

[1] Editas Med, Cambridge, MA USA

来源：

HUMAN GENE THERAPY | 2015年 / 26卷 / 07期

关键词：

DEFECTIVE LENTIVIRAL VECTORS; OFF-TARGET CLEAVAGE; ONE-STEP GENERATION; HEPATITIS-B-VIRUS; HUMAN-CELLS; ADENOASSOCIATED VIRUS; NUCLEASE SPECIFICITY; CAS NUCLEASES; HEMATOPOIETIC STEM; NONDIVIDING CELLS;

D O I：

10.1089/hum.2015.074

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 [微生物学]; 090105 [作物生产系统与生态工程];

摘要：

Genome editing using the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR associated 9 (Cas9) technology is revolutionizing the study of gene function and likely will give rise to an entire new class of therapeutics for a wide range of diseases. Achieving this goal requires not only characterization of the technology for efficacy and specificity but also optimization of its delivery to the target cells for each disease indication. In this review we survey the various methods by which the CRISPR/Cas9 components have been delivered to cells and highlight some of the more clinically relevant approaches. Additionally, we discuss the methods available for assessing the specificity of Cas9 editing; an important safety consideration for development of the technology.

引用

页码：443 / 451

页数：9

共 95 条

[1]

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome [J].

Aiuti, Alessandro ;

Biasco, Luca ;

Scaramuzza, Samantha ;

Ferrua, Francesca ;

Cicalese, Maria Pia ;

Baricordi, Cristina ;

Dionisio, Francesca ;

Calabria, Andrea ;

Giannelli, Stefania ;

Castiello, Maria Carmina ;

Bosticardo, Marita ;

Evangelio, Costanza ;

Assanelli, Andrea ;

Casiraghi, Miriam ;

Di Nunzio, Sara ;

Callegaro, Luciano ;

Benati, Claudia ;

Rizzardi, Paolo ;

Pellin, Danilo ;

Di Serio, Clelia ;

Schmidt, Manfred ;

Von Kalle, Christof ;

Gardner, Jason ;

Mehta, Nalini ;

Neduva, Victor ;

Dow, David J. ;

Galy, Anne ;

Miniero, Roberto ;

Finocchi, Andrea ;

Metin, Ayse ;

Banerjee, Pinaki P. ;

Orange, Jordan S. ;

Galimberti, Stefania ;

Valsecchi, Maria Grazia ;

Biffi, Alessandra ;

Montini, Eugenio ;

Villa, Anna ;

Ciceri, Fabio ;

Roncarolo, Maria Grazia ;

Naldini, Luigi .

SCIENCE, 2013, 341 (6148) :865-U71

[2]

Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice [J].

Allocca, Mariacarmela ;

Doria, Monica ;

Petrillo, Marco ;

Colella, Pasqualina ;

Garcia-Hoyos, Maria ;

Gibbs, Daniel ;

Kim, So Ra ;

Maguire, Albert ;

Rex, Tonia S. ;

Di Vicino, Umberto ;

Cutillo, Luisa ;

Sparrow, Janet R. ;

Williams, David S. ;

Bennett, Jean ;

Auricchio, Alberto .

JOURNAL OF CLINICAL INVESTIGATION, 2008, 118 (05) :1955-1964

[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]

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy [J].

Biffi, Alessandra ;

Montini, Eugenio ;

Lorioli, Laura ;

Cesani, Martina ;

Fumagalli, Francesca ;

Plati, Tiziana ;

Baldoli, Cristina ;

Martino, Sabata ;

Calabria, Andrea ;

Canale, Sabrina ;

Benedicenti, Fabrizio ;

Vallanti, Giuliana ;

Biasco, Luca ;

Leo, Simone ;

Kabbara, Nabil ;

Zanetti, Gianluigi ;

Rizzo, William B. ;

Mehta, Nalini A. L. ;

Cicalese, Maria Pia ;

Casiraghi, Miriam ;

Boelens, Jaap J. ;

Del Carro, Ubaldo ;

Dow, David J. ;

Schmidt, Manfred ;

Assanelli, Andrea ;

Neduva, Victor ;

Di Serio, Clelia ;

Stupka, Elia ;

Gardner, Jason ;

von Kalle, Christof ;

Bordignon, Claudio ;

Ciceri, Fabio ;

Rovelli, Attilio ;

Roncarolo, Maria Grazia ;

Aiuti, Alessandro ;

Sessa, Maria ;

Naldini, Luigi .

SCIENCE, 2013, 341 (6148) :864-U58

[5]

Exploiting CRISPR-Cas nucleases to produce sequence-specific antimicrobials [J].

Bikard, David ;

Euler, Chad W. ;

Jiang, Wenyan ;

Nussenzweig, Philip M. ;

Goldberg, Gregory W. ;

Duportet, Xavier ;

Fischetti, Vincent A. ;

Marraffini, Luciano A. .

NATURE BIOTECHNOLOGY, 2014, 32 (11) :1146-1150

[6]

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

[7]

Burnett James C, 2010, Open Access Bioinformatics, V2010, P11

[8]

Staying on target with CRISPR-Cas [J].

Carroll, Dana .

NATURE BIOTECHNOLOGY, 2013, 31 (09) :807-809

[9]

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy [J].

Cartier, Nathalie ;

Hacein-Bey-Abina, Salima ;

Bartholomae, Cynthia C. ;

Veres, Gabor ;

Schmidt, Manfred ;

Kutschera, Ina ;

Vidaud, Michel ;

Abel, Ulrich ;

Dal-Cortivo, Liliane ;

Caccavelli, Laure ;

Mahlaoui, Nizar ;

Kiermer, Veronique ;

Mittelstaedt, Denice ;

Bellesme, Celine ;

Lahlou, Najiba ;

Lefrere, Francois ;

Blanche, Stephane ;

Audit, Muriel ;

Payen, Emmanuel ;

Leboulch, Philippe ;

l'Homme, Bruno ;

Bougneres, Pierre ;

Von Kalle, Christof ;

Fischer, Alain ;

Cavazzana-Calvo, Marina ;

Aubourg, Patrick .

SCIENCE, 2009, 326 (5954) :818-823

[10]

Genome-wide Translocation Sequencing Reveals Mechanisms of Chromosome Breaks and Rearrangements in B Cells [J].

Chiarle, Roberto ;

Zhang, Yu ;

Frock, Richard L. ;

Lewis, Susanna M. ;

Molinie, Benoit ;

Ho, Yu-Jui ;

Myers, Darienne R. ;

Choi, Vivian W. ;

Compagno, Mara ;

Malkin, Daniel J. ;

Neuberg, Donna ;

Monti, Stefano ;

Giallourakis, Cosmas C. ;

Gostissa, Monica ;

Alt, Frederick W. .

CELL, 2011, 147 (01) :107-119

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