Engineering Delivery Vehicles for Genome Editing

被引：95

作者：

Nelson, Christopher E. ^{[1
,2
]}

Gersbach, Charles A. ^{[1
,2
,3
]}

机构：

[1] Duke Univ, Dept Biomed Engn, Durham, NC 27708 USA

[2] Duke Univ, Ctr Genom & Computat Biol, Durham, NC 27708 USA

[3] Duke Univ, Dept Orthopaed Surg, Durham, NC 27708 USA

来源：

ANNUAL REVIEW OF CHEMICAL AND BIOMOLECULAR ENGINEERING, VOL 7 | 2016年 / 7卷

关键词：

viral gene therapy; nonviral gene therapy; zinc finger nucleases; TALENs; CRISPR; nanoparticle; virus; DUCHENNE MUSCULAR-DYSTROPHY; ZINC-FINGER NUCLEASES; HEMATOPOIETIC STEM/PROGENITOR CELLS; SEQUENCE-SPECIFIC ANTIMICROBIALS; ADENOASSOCIATED VIRUS AAV; RNA-GUIDED ENDONUCLEASE; ENHANCED GENE DELIVERY; MODIFIED MESSENGER-RNA; DOUBLE-STRAND BREAKS; CD4(+) T-CELLS;

D O I：

10.1146/annurev-chembioeng-080615-034711

中图分类号：

O69 [应用化学];

学科分类号：

081704 ;

摘要：

The field of genome engineering has created new possibilities for gene therapy, including improved animal models of disease, engineered cell therapies, and in vivo gene repair. The most significant challenge for the clinical translation of genome engineering is the development of safe and effective delivery vehicles. A large body of work has applied genome engineering to genetic modification in vitro, and clinical trials have begun using cells modified by genome editing. Now, promising preclinical work is beginning to apply these tools in vivo. This article summarizes the development of genome engineering platforms, including meganucleases, zinc finger nucleases, TALENs, and CRISPR/Cas9, and their flexibility for precise genetic modifications. The prospects for the development of safe and effective viral and nonviral delivery vehicles for genome editing are reviewed, and promising advances in particular therapeutic applications are discussed.

引用

页码：637 / 662

页数：26

共 185 条

[1] Exploring polyethylenimine-mediated DNA transfection and the proton sponge hypothesis [J].

Akinc, A ;

Thomas, M ;

Klibanov, AM ;

Langer, R .

JOURNAL OF GENE MEDICINE, 2005, 7 (05) :657-663

[2] A combinatorial library of lipid-like materials for delivery of RNAi therapeutics [J].

Akinc, Akin ;

Zumbuehl, Andreas ;

Goldberg, Michael ;

Leshchiner, Elizaveta S. ;

Busini, Valentina ;

Hossain, Naushad ;

Bacallado, Sergio A. ;

Nguyen, David N. ;

Fuller, Jason ;

Alvarez, Rene ;

Borodovsky, Anna ;

Borland, Todd ;

Constien, Rainer ;

de Fougerolles, Antonin ;

Dorkin, J. Robert ;

Jayaprakash, K. Narayanannair ;

Jayaraman, Muthusamy ;

John, Matthias ;

Koteliansky, Victor ;

Manoharan, Muthiah ;

Nechev, Lubomir ;

Qin, June ;

Racie, Timothy ;

Raitcheva, Denitza ;

Rajeev, Kallanthottathil G. ;

Sah, Dinah W. Y. ;

Soutschek, Juergen ;

Toudjarska, Ivanka ;

Vornlocher, Hans-Peter ;

Zimmermann, Tracy S. ;

Langer, Robert ;

Anderson, Daniel G. .

NATURE BIOTECHNOLOGY, 2008, 26 (05) :561-569

[3] Chimeric recombinases with designed DNA sequence recognition [J].

Akopian, A ;

He, JY ;

Boocock, MR ;

Stark, WM .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2003, 100 (15) :8688-8691

[4] Factors affecting the clearance and biodistribution of polymeric nanoparticles [J].

Alexis, Frank ;

Pridgen, Eric ;

Molnar, Linda K. ;

Farokhzad, Omid C. .

MOLECULAR PHARMACEUTICS, 2008, 5 (04) :505-515

[5] Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial [J].

Alton, Eric W. F. W. ;

Armstrong, David K. ;

Ashby, Deborah ;

Bayfield, Katie J. ;

Bilton, Diana ;

Bloomfield, Emily V. ;

Boyd, A. Christopher ;

Brand, June ;

Buchan, Ruaridh ;

Calcedo, Roberto ;

Carvelli, Paula ;

Chan, Mario ;

Cheng, Seng H. ;

Collie, D. David S. ;

Cunningham, Steve ;

Davidson, Heather E. ;

Davies, Gwyneth ;

Davies, Jane C. ;

Davies, Lee A. ;

Dewar, Maria H. ;

Doherty, Ann ;

Donovan, Jackie ;

Dwyer, Natalie S. ;

Elgmati, Hala I. ;

Featherstone, Rosanna F. ;

Gavino, Jemyr ;

Gea-Sorli, Sabrina ;

Geddes, Duncan M. ;

Gibson, James S. R. ;

Gill, Deborah R. ;

Greening, Andrew P. ;

Griesenbach, Uta ;

Hansell, David M. ;

Harman, Katharine ;

Higgins, Tracy E. ;

Hodges, Samantha L. ;

Hyde, Stephen C. ;

Hyndman, Laura ;

Innes, J. Alastair ;

Jacob, Joseph ;

Jones, Nancy ;

Keogh, Brian F. ;

Limberis, Maria P. ;

Lloyd-Evans, Paul ;

Maclean, Alan W. ;

Manvell, Michelle C. ;

McCormick, Dominique ;

McGovern, Michael ;

McLachlan, Gerry ;

Meng, Cuixiang .

LANCET RESPIRATORY MEDICINE, 2015, 3 (09) :684-691

[6] 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

[7] Engineering of large numbers of highly specific homing endonucleases that induce recombination on novel DNA targets [J].

Arnould, S ;

Chames, P ;

Perez, C ;

Lacroix, E ;

Duclert, A ;

Epinat, JC ;

Stricher, F ;

Petit, AS ;

Patin, A ;

Guillier, S ;

Rolland, S ;

Prieto, J ;

Blanco, FJ ;

Bravo, J ;

Montoya, G ;

Serrano, L ;

Duchateau, P ;

Pâques, F .

JOURNAL OF MOLECULAR BIOLOGY, 2006, 355 (03) :443-458

[8] Computational redesign of endonuclease DNA binding and cleavage specificity [J].

Ashworth, Justin ;

Havranek, James J. ;

Duarte, Carlos M. ;

Sussman, Django ;

Monnat, Raymond J., Jr. ;

Stoddard, Barry L. ;

Baker, David .

NATURE, 2006, 441 (7093) :656-659

[9] Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle [J].

Asokan, Aravind ;

Conway, Julia C. ;

Phillips, Jana L. ;

Li, Chengwen ;

Hegge, Julia ;

Sinnott, Rebecca ;

Yadav, Swati ;

DiPrimio, Nina ;

Nam, Hyun-Joo ;

Agbandje-McKenna, Mavis ;

McPhee, Scott ;

Wolff, Jon ;

Samulski, R. Jude .

NATURE BIOTECHNOLOGY, 2010, 28 (01) :79-U107

[10] 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

← 1 2 3 4 5 6 7 8 9 10 →