Genetic therapy - Past, present, and future

被引:12
作者
Flotte, TR
Ferkol, TW
机构
[1] UNIV FLORIDA, DEPT MOL GENET & MICROBIOL, SCH MED, GAINESVILLE, FL 32610 USA
[2] UNIV FLORIDA, GENE THERAPY CTR, SCH MED, GAINESVILLE, FL 32610 USA
[3] CASE WESTERN RESERVE UNIV, RAINBOW BABIES & CHILDRENS HOSP,SCH MED, DEPT PEDIAT,DIV PEDIAT PULM, CLEVELAND, OH 44106 USA
关键词
D O I
10.1016/S0031-3955(05)70468-5
中图分类号
R72 [儿科学];
学科分类号
100202 ;
摘要
Because of its promise, few fields of biomedical research have captured the public's imagination as has genetic therapy. Genetic therapy has the potential to prevent, correct, or ameliorate various genetic and nongenetic conditions, provided that methods of delivering DNA to affected cells are safe, efficient, and selective. Yet these technologies are still in their infancy, and although the early results of some clinical trials are encouraging, no one has yet been cured with genetic therapy. Progress in the field is Limited by the shortcomings of the available vectors used for gene transfer. The development and improvement of viral and nonviral vectors may allow them to reach their potential and serve as alternative therapies for disease.
引用
收藏
页码:153 / +
页数:1
相关论文
共 182 条
[1]  
ACSADI G, 1991, NEW BIOL, V3, P71
[2]   HUMAN DYSTROPHIN EXPRESSION IN MDX MICE AFTER INTRAMUSCULAR INJECTION OF DNA CONSTRUCTS [J].
ACSADI, G ;
DICKSON, G ;
LOVE, DR ;
JANI, A ;
WALSH, FS ;
GURUSINGHE, A ;
WOLFF, JA ;
DAVIES, KE .
NATURE, 1991, 352 (6338) :815-818
[3]   In vivo model of adeno-associated virus vector persistence and rescue [J].
Afione, SA ;
Conrad, CK ;
Kearns, WG ;
Chunduru, S ;
Adams, R ;
Reynolds, TC ;
Guggino, WB ;
Cutting, GR ;
Carter, BJ ;
Flotte, TR .
JOURNAL OF VIROLOGY, 1996, 70 (05) :3235-3241
[4]   LONG-CIRCULATING (STERICALLY STABILIZED) LIPOSOMES FOR TARGETED DRUG-DELIVERY [J].
ALLEN, TM .
TRENDS IN PHARMACOLOGICAL SCIENCES, 1994, 15 (07) :215-220
[5]   NONINVASIVE LIPOSOME-MEDIATED GENE DELIVERY CAN CORRECT THE ION-TRANSPORT DEFECT IN CYSTIC-FIBROSIS MUTANT MICE [J].
ALTON, EWFW ;
MIDDLETON, PG ;
CAPLEN, NJ ;
SMITH, SN ;
STEEL, DM ;
MUNKONGE, FM ;
JEFFERY, PK ;
GEDDES, DM ;
HART, SL ;
WILLIAMSON, R ;
FASOLD, KI ;
MILLER, AD ;
DICKINSON, P ;
STEVENSON, BJ ;
MCLACHLAN, G ;
DORIN, JR ;
PORTEOUS, DJ .
NATURE GENETICS, 1993, 5 (02) :135-142
[6]  
[Anonymous], 1990, Fields Virology
[7]  
[Anonymous], 1990, VIROLOGY
[8]   PHENOTYPIC CORRECTION OF FACTOR-IX DEFICIENCY IN SKIN FIBROBLASTS OF HEMOPHILIC DOGS [J].
AXELROD, JH ;
READ, MS ;
BRINKHOUS, KM ;
VERMA, IM .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1990, 87 (13) :5173-5177
[9]   IN-VITRO EVIDENCE THAT METABOLIC COOPERATION IS RESPONSIBLE FOR THE BYSTANDER EFFECT OBSERVED WITH HSV TK RETROVIRAL GENE-THERAPY [J].
BI, WL ;
PARYSEK, LM ;
WARNICK, R ;
STAMBROOK, PJ .
HUMAN GENE THERAPY, 1993, 4 (06) :725-731