Gene therapy in clinical medicine

被引:39
作者
Selkirk, SM [1 ]
机构
[1] Univ Hosp Cleveland, Dept Neurol, Cleveland, OH 44106 USA
关键词
D O I
10.1136/pgmj.2003.017764
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Although the field of gene therapy has experienced significant setbacks and limited success, it is one of the most promising and active research fields in medicine. Interest in this therapeutic modality is based on the potential for treatment and cure of some of the most malignant and devastating diseases affecting humans. Over the next decade, the relevance of gene therapy to medical practices will increase and it will become important for physicians to understand the basic principles and strategies that underlie the therapeutic intervention. This report reviews the history, basic strategies, tools, and several current clinical paradigms for application.
引用
收藏
页码:560 / 570
页数:11
相关论文
共 159 条
[1]   Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients [J].
Aartsma-Rus, A ;
Janson, AAM ;
Kaman, WE ;
Bremmer-Bout, M ;
den Dunnen, JT ;
Baas, F ;
van Ommen, GJB ;
van Deutekom, JCT .
HUMAN MOLECULAR GENETICS, 2003, 12 (08) :907-914
[2]   Lentiviral-mediated RNA interference [J].
Abbas-Terki, T ;
Blanco-Bose, W ;
Déglon, N ;
Pralong, W ;
Aebischer, P .
HUMAN GENE THERAPY, 2002, 13 (18) :2197-2201
[3]   A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease [J].
Aitken, ML ;
Moss, RB ;
Waltz, DA ;
Dovey, ME ;
Tonelli, MR ;
McNamara, SC ;
Gibson, RL ;
Ramsey, BW ;
Carter, BJ ;
Reynolds, TC .
HUMAN GENE THERAPY, 2001, 12 (15) :1907-1916
[4]   Biomedicine - Lentiviral vectors - the promise of gene therapy within reach? [J].
Amado, RG ;
Chen, ISY .
SCIENCE, 1999, 285 (5428) :674-676
[5]   PROSPECTS FOR HUMAN-GENE THERAPY [J].
ANDERSON, WF .
SCIENCE, 1984, 226 (4673) :401-409
[6]   HUMAN GENE-THERAPY [J].
ANDERSON, WF .
SCIENCE, 1992, 256 (5058) :808-813
[7]   GENE-THERAPY IN HUMAN-BEINGS - WHEN IS IT ETHICAL TO BEGIN [J].
ANDERSON, WF ;
FLETCHER, JC .
NEW ENGLAND JOURNAL OF MEDICINE, 1980, 303 (22) :1293-1297
[8]   Engraftment of autologous retrovirally transduced hepatocytes after intraportal transplantation into nonhuman primates:: Implication for ex vivo gene therapy [J].
Andreoletti, M ;
Loux, N ;
Vons, C ;
Nguyen, TH ;
Lorand, I ;
Mahieu, D ;
Simon, L ;
Di Rico, V ;
Vingert, B ;
Chapman, J ;
Briand, P ;
Schwall, R ;
Hamza, J ;
Capron, F ;
Bargy, F ;
Franco, D ;
Weber, A .
HUMAN GENE THERAPY, 2001, 12 (02) :169-179
[9]   ANGIOGENIC-INDUCED ENHANCEMENT OF COLLATERAL BLOOD-FLOW TO ISCHEMIC MYOCARDIUM BY VASCULAR ENDOTHELIAL GROWTH-FACTOR IN DOGS [J].
BANAI, S ;
JAKLITSCH, MT ;
SHOU, M ;
LAZAROUS, DF ;
SCHEINOWITZ, M ;
BIRO, S ;
EPSTEIN, SE ;
UNGER, EF .
CIRCULATION, 1994, 89 (05) :2183-2189
[10]   RNA interference - The short answer [J].
Bass, BL .
NATURE, 2001, 411 (6836) :428-429