Progress and problems with the use of viral vectors for gene therapy

被引:1961
作者
Thomas, CE
Ehrhardt, A
Kay, MA [1 ]
机构
[1] Stanford Univ, Sch Med, Dept Pediat, Stanford, CA 94305 USA
[2] Stanford Univ, Sch Med, Dept Genet, Stanford, CA 94305 USA
基金
美国国家卫生研究院;
关键词
D O I
10.1038/nrg1066
中图分类号
Q3 [遗传学];
学科分类号
071007 ; 090102 ;
摘要
Gene therapy has a history of controversy. Encouraging results are starting to emerge from the clinic, but questions are still being asked about the safety of this new molecular medicine. With the development of a leukaemia-like syndrome in two of the small number of patients that have been cured of a disease by gene therapy, it is timely to contemplate how far this technology has come, and how far it still has to go.
引用
收藏
页码:346 / 358
页数:13
相关论文
共 121 条
[71]   Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver [J].
Nakai, H ;
Iwaki, Y ;
Kay, MA ;
Couto, LB .
JOURNAL OF VIROLOGY, 1999, 73 (07) :5438-5447
[72]   Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector [J].
Naldini, L ;
Blomer, U ;
Gage, FH ;
Trono, D ;
Verma, IM .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1996, 93 (21) :11382-11388
[73]  
Nemunaitis J, 2000, CANCER RES, V60, P6359
[74]   Toxicological comparison of E2a-deleted and first-generation adenoviral vectors expressing α1-antitrypsin after systemic delivery [J].
O'Neal, WK ;
Zhou, HS ;
Morral, N ;
Aguilar-Cordova, E ;
Pestaner, J ;
Langston, C ;
Mull, B ;
Wang, YF ;
Beaudet, AL ;
Lee, B .
HUMAN GENE THERAPY, 1998, 9 (11) :1587-1598
[75]   Site-specific genomic integration produces therapeutic Factor IX levels in mice [J].
Olivares, EC ;
Hollis, RP ;
Chalberg, TW ;
Meuse, L ;
Kay, MA ;
Calos, MP .
NATURE BIOTECHNOLOGY, 2002, 20 (11) :1124-1128
[76]   Stable nonviral genetic correction of inherited human skin disease [J].
Ortiz-Urda, S ;
Thyagarajan, B ;
Keene, DR ;
Lin, Q ;
Fang, M ;
Calos, MP ;
Khavari, PA .
NATURE MEDICINE, 2002, 8 (10) :1166-1170
[77]   Negative-strand RNA viruses: Genetic engineering and applications [J].
Palese, P ;
Zheng, HY ;
Engelhardt, OG ;
Pleschka, S ;
GarciaSastre, A .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1996, 93 (21) :11354-11358
[78]   Development and optimization of herpes simplex virus vectors for multiple long-term gene delivery to the peripheral nervous system [J].
Palmer, JA ;
Branston, RH ;
Lilley, CE ;
Robinson, MJ ;
Groutsi, F ;
Smith, J ;
Latchman, DS ;
Coffin, RS .
JOURNAL OF VIROLOGY, 2000, 74 (12) :5604-5618
[79]   Silencing of gene expression: implications for design of retrovirus vectors [J].
Pannell, D ;
Ellis, J .
REVIEWS IN MEDICAL VIROLOGY, 2001, 11 (04) :205-217
[80]   Efficient lentiviral transduction of liver requires cell cycling in vivo [J].
Park, F ;
Ohashi, K ;
Chiu, W ;
Naldini, L ;
Kay, MA .
NATURE GENETICS, 2000, 24 (01) :49-52