Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications

被引：84
作者：
Gaj, Thomas [1 ]
Epstein, Benjamin E. [2 ]
Schaffer, David V. [1 ,2 ,3 ,4 ]
机构：
[1] Univ Calif Berkeley, Dept Chem & Biomol Engn, Berkeley, CA 94720 USA
[2] Univ Calif Berkeley, Dept Bioengn, Berkeley, CA 94720 USA
[3] Univ Calif Berkeley, Dept Cell & Mol Biol, Berkeley, CA 94720 USA
[4] Univ Calif Berkeley, Helen Wills Neurosci Inst, Berkeley, CA 94720 USA
来源：
MOLECULAR THERAPY | 2016年 / 24卷 / 03期
基金：
美国国家卫生研究院;
关键词：
SITE-SPECIFIC INTEGRATION; HEPARAN-SULFATE PROTEOGLYCAN; IN-VIVO; HOMOLOGOUS RECOMBINATION; VECTOR INTEGRATION; GENE-THERAPY; TARGETED INTEGRATION; DNA-SEQUENCES; MOUSE MODEL; STEM-CELLS;
D O I：
10.1038/mt.2015.151
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 ; 0836 ; 090102 ; 100705 ;
摘要：
In addition to their broad potential for therapeutic gene delivery, adeno-associated virus (AAV) vectors possess the innate ability to stimulate homologous recombination in mammalian cells at high efficiencies. This process referred to as AAV-mediated gene targeting has enabled the introduction of a diverse array of genomic modifications both in vitro and in vivo. With the recent emergence of targeted nucleases, AAV-mediated genome engineering is poised for clinical translation. Here, we review key properties of AAV vectors that underscore its unique utility in genome editing. We highlight the broad range of genome engineering applications facilitated by this technology and discuss the strong potential for unifying AAV with targeted nucleases for next-generation gene therapy.
引用
收藏
页码：458 / 464
页数：7
相关论文
共 107 条
  • [1] Directed Evolution of Adeno-associated Virus for Enhanced Gene Delivery and Gene Targeting in Human Pluripotent Stem Cells
    Asuri, Prashanth
    Bartel, Melissa A.
    Vazin, Tandis
    Jang, Jae-Hyung
    Wong, Tiffany B.
    Schaffer, David V.
    [J]. MOLECULAR THERAPY, 2012, 20 (02) : 329 - 338
  • [2] Effect of gene therapy on visual function in Leber's congenital amaurosis
    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.
    [J]. NEW ENGLAND JOURNAL OF MEDICINE, 2008, 358 (21) : 2231 - 2239
  • [3] Infectious entry pathway of adeno-associated virus and adeno-associated virus vectors
    Bartlett, JS
    Wilcher, R
    Samulski, RJ
    [J]. JOURNAL OF VIROLOGY, 2000, 74 (06) : 2777 - 2785
  • [4] Promoter less gene targeting without nucleases ameliorates haemophilia B in mice
    Barzel, A.
    Paulk, N. K.
    Shi, Y.
    Huang, Y.
    Chu, K.
    Zhang, F.
    Valdmanis, P. N.
    Spector, L. P.
    Porteus, M. H.
    Gaensler, K. M.
    Kay, M. A.
    [J]. NATURE, 2015, 517 (7534) : 360 - U476
  • [5] AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness
    Bennett, Jean
    Ashtari, Manzar
    Wellman, Jennifer
    Marshall, Kathleen A.
    Cyckowski, Laura L.
    Chung, Daniel C.
    McCague, Sarah
    Pierce, Eric A.
    Chen, Yifeng
    Bennicelli, Jeannette L.
    Zhu, Xiaosong
    Ying, Gui-shuang
    Sun, Junwei
    Wright, J. Fraser
    Auricchio, Alberto
    Simonelli, Francesca
    Shindler, Kenneth S.
    Mingozzi, Federico
    High, Katherine A.
    Maguire, Albert M.
    [J]. SCIENCE TRANSLATIONAL MEDICINE, 2012, 4 (120)
  • [6] Stimulation of homologous recombination through targeted cleavage by chimeric nucleases
    Bibikova, M
    Carroll, D
    Segal, DJ
    Trautman, JK
    Smith, J
    Kim, YG
    Chandrasegaran, S
    [J]. MOLECULAR AND CELLULAR BIOLOGY, 2001, 21 (01) : 289 - 297
  • [7] Genome-wide association study of 14,000 cases of seven common diseases and 3,000 shared controls
    Burton, Paul R.
    Clayton, David G.
    Cardon, Lon R.
    Craddock, Nick
    Deloukas, Panos
    Duncanson, Audrey
    Kwiatkowski, Dominic P.
    McCarthy, Mark I.
    Ouwehand, Willem H.
    Samani, Nilesh J.
    Todd, John A.
    Donnelly, Peter
    Barrett, Jeffrey C.
    Davison, Dan
    Easton, Doug
    Evans, David
    Leung, Hin-Tak
    Marchini, Jonathan L.
    Morris, Andrew P.
    Spencer, Chris C. A.
    Tobin, Martin D.
    Attwood, Antony P.
    Boorman, James P.
    Cant, Barbara
    Everson, Ursula
    Hussey, Judith M.
    Jolley, Jennifer D.
    Knight, Alexandra S.
    Koch, Kerstin
    Meech, Elizabeth
    Nutland, Sarah
    Prowse, Christopher V.
    Stevens, Helen E.
    Taylor, Niall C.
    Walters, Graham R.
    Walker, Neil M.
    Watkins, Nicholas A.
    Winzer, Thilo
    Jones, Richard W.
    McArdle, Wendy L.
    Ring, Susan M.
    Strachan, David P.
    Pembrey, Marcus
    Breen, Gerome
    St Clair, David
    Caesar, Sian
    Gordon-Smith, Katherine
    Jones, Lisa
    Fraser, Christine
    Green, Elain K.
    [J]. NATURE, 2007, 447 (7145) : 661 - 678
  • [8] Effect of Alipogene Tiparvovec (AAV1-LPLS447X) on Postprandial Chylomicron Metabolism in Lipoprotein Lipase-Deficient Patients
    Carpentier, Andre C.
    Frisch, Frederique
    Labbe, Sebastien M.
    Gagnon, Rene
    de Wal, Janneke
    Greentree, Stephen
    Petry, Harald
    Twisk, Jaap
    Brisson, Diane
    Gaudet, Daniel
    [J]. JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM, 2012, 97 (05) : 1635 - 1644
  • [9] Gene targeting in stem cells from individuals with osteogenesis imperfecta
    Chamberlain, JR
    Schwarze, U
    Wang, PR
    Hirata, RK
    Hankenson, KD
    Pace, JM
    Underwood, RA
    Song, KM
    Sussman, M
    Byers, PH
    Russell, DW
    [J]. SCIENCE, 2004, 303 (5661) : 1198 - 1201
  • [10] Multiplex Genome Engineering Using CRISPR/Cas Systems
    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
    [J]. SCIENCE, 2013, 339 (6121) : 819 - 823
12345678910