Directed evolution of novel adeno-associated viruses for therapeutic gene delivery

被引:126
作者
Bartel, M. A. [1 ]
Weinstein, J. R. [2 ]
Schaffer, D. V. [1 ,3 ,4 ]
机构
[1] Univ Calif Berkeley, Dept Chem & Biomol Engn, Berkeley, CA 94720 USA
[2] Univ Calif Berkeley, Dept Chem, Berkeley, CA 94720 USA
[3] Univ Calif Berkeley, Dept Bioengn, Berkeley, CA 94720 USA
[4] Univ Calif Berkeley, Helen Wills Neurosci Inst, Berkeley, CA 94720 USA
关键词
directed evolution; adeno-associated virus; viral vector; gene delivery; gene targeting; TRANSIENT IMMUNOSUPPRESSION; PEPTIDE LIBRARIES; VIRAL VARIANT; IN-VITRO; PHASE-I; AAV; VECTORS; TRANSDUCTION; EFFICIENT; GENERATION;
D O I
10.1038/gt.2012.20
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
Gene therapy vectors based on adeno-associated virus (AAV) are currently in clinical trials for numerous disease targets, such as muscular dystrophy, hemophilia, Parkinson's disease, Leber's congenital amaurosis and macular degeneration. Despite its considerable promise and emerging clinical success, several challenges impede the broader implementation of AAV gene therapy, including the prevalence of neutralizing antibodies in the human population, low transduction of a number of therapeutically relevant cell and tissue types, an inability to overcome physical and cellular barriers in vivo and a relatively limited carrying capacity. These challenges arise as the demands we place on AAV vectors are often different from or even at odds with the properties nature bestowed on their parent viruses. Viral-directed evolution-the iterative generation of large, diverse libraries of viral mutants and selection for variants with specific properties of interest-offers an approach to address these problems. Here we outline progress in creating novel classes of AAV variant libraries and highlight the successful isolation of variants with novel and advantageous in vitro and in vivo gene delivery properties. Gene Therapy (2012) 19, 694-700; doi:10.1038/gt.2012.20; published online 8 March 2012
引用
收藏
页码:694 / 700
页数:7
相关论文
共 65 条
[1]   Directed Evolution of Adeno-associated Virus for Enhanced Gene Delivery and Gene Targeting in Human Pluripotent Stem Cells [J].
Asuri, Prashanth ;
Bartel, Melissa A. ;
Vazin, Tandis ;
Jang, Jae-Hyung ;
Wong, Tiffany B. ;
Schaffer, David V. .
MOLECULAR THERAPY, 2012, 20 (02) :329-338
[2]   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
[3]   Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors [J].
Boutin, Sylvie ;
Monteilhet, Virginie ;
Veron, Philippe ;
Leborgne, Christian ;
Benveniste, Olivier ;
Montus, Marie Francoise ;
Masurier, Carole .
HUMAN GENE THERAPY, 2010, 21 (06) :704-712
[4]   Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector [J].
Bowles, Dawn E. ;
McPhee, Scott W. J. ;
Li, Chengwen ;
Gray, Steven J. ;
Samulski, Jade J. ;
Camp, Angelique S. ;
Li, Juan ;
Wang, Bing ;
Monahan, Paul E. ;
Rabinowitz, Joseph E. ;
Grieger, Joshua C. ;
Govindasamy, Lakshmanan ;
Agbandje-McKenna, Mavis ;
Xiao, Xiao ;
Samulski, R. Jude .
MOLECULAR THERAPY, 2012, 20 (02) :443-455
[5]   Marker rescue of adeno-associated virus (AAV) capsid mutants: a novel approach for chimeric AAV production [J].
Bowles, DE ;
Rabinowitz, JE ;
Samulski, RJ .
JOURNAL OF VIROLOGY, 2003, 77 (01) :423-432
[6]   Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease [J].
Cabrera-Salazar, Mario A. ;
Roskelley, Eric M. ;
Bu, Jie ;
Hodges, Bradley L. ;
Yew, Nelson ;
Dodge, James C. ;
Shihabuddin, Lamya S. ;
Sohar, Istvan ;
Sleat, David E. ;
Scheule, Ronald K. ;
Davidson, Beverly L. ;
Cheng, Seng H. ;
Lobel, Peter ;
Passini, Marco A. .
MOLECULAR THERAPY, 2007, 15 (10) :1782-1788
[7]   Worldwide Epidemiology of Neutralizing Antibodies to Adeno-Associated Viruses [J].
Calcedo, Roberto ;
Vandenberghe, Luk H. ;
Gao, Guangping ;
Lin, Jianping ;
Wilson, James M. .
JOURNAL OF INFECTIOUS DISEASES, 2009, 199 (03) :381-390
[8]  
Dalkara D, 2011, AM SOC GEN CELL THER
[9]   AAV Mediated GDNF Secretion From Retinal Glia Slows Down Retinal Degeneration in a Rat Model of Retinitis Pigmentosa [J].
Dalkara, Deniz ;
Kolstad, Kathleen D. ;
Guerin, Karen I. ;
Hoffmann, Natalie V. ;
Visel, Meike ;
Klimczak, Ryan R. ;
Schaffer, David V. ;
Flannery, John G. .
MOLECULAR THERAPY, 2011, 19 (09) :1602-1608
[10]   Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue [J].
Duan, DS ;
Sharma, P ;
Yang, JS ;
Yue, YP ;
Dudus, L ;
Zhang, YL ;
Fisher, KJ ;
Engelhardt, JF .
JOURNAL OF VIROLOGY, 1998, 72 (11) :8568-8577