共 159 条
Gene therapy in clinical medicine
被引:39
作者:

Selkirk, SM
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Cleveland, Dept Neurol, Cleveland, OH 44106 USA Univ Hosp Cleveland, Dept Neurol, Cleveland, OH 44106 USA
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

Aartsma-Rus, A
论文数: 0 引用数: 0
h-index: 0
机构: Leiden Univ, Med Ctr, Ctr Human & Clin Genet, NL-2333 AL Leiden, Netherlands

Janson, AAM
论文数: 0 引用数: 0
h-index: 0
机构: Leiden Univ, Med Ctr, Ctr Human & Clin Genet, NL-2333 AL Leiden, Netherlands

Kaman, WE
论文数: 0 引用数: 0
h-index: 0
机构: Leiden Univ, Med Ctr, Ctr Human & Clin Genet, NL-2333 AL Leiden, Netherlands

Bremmer-Bout, M
论文数: 0 引用数: 0
h-index: 0
机构: Leiden Univ, Med Ctr, Ctr Human & Clin Genet, NL-2333 AL Leiden, Netherlands

den Dunnen, JT
论文数: 0 引用数: 0
h-index: 0
机构: Leiden Univ, Med Ctr, Ctr Human & Clin Genet, NL-2333 AL Leiden, Netherlands

Baas, F
论文数: 0 引用数: 0
h-index: 0
机构: Leiden Univ, Med Ctr, Ctr Human & Clin Genet, NL-2333 AL Leiden, Netherlands

van Ommen, GJB
论文数: 0 引用数: 0
h-index: 0
机构: Leiden Univ, Med Ctr, Ctr Human & Clin Genet, NL-2333 AL Leiden, Netherlands

van Deutekom, JCT
论文数: 0 引用数: 0
h-index: 0
机构: Leiden Univ, Med Ctr, Ctr Human & Clin Genet, NL-2333 AL Leiden, Netherlands
[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

Abbas-Terki, T
论文数: 0 引用数: 0
h-index: 0
机构:
Ecole Polytech Fed Lausanne, Swiss Fed Inst Technol, Inst Neurosci, CH-1015 Lausanne, Switzerland Ecole Polytech Fed Lausanne, Swiss Fed Inst Technol, Inst Neurosci, CH-1015 Lausanne, Switzerland

Blanco-Bose, W
论文数: 0 引用数: 0
h-index: 0
机构:
Ecole Polytech Fed Lausanne, Swiss Fed Inst Technol, Inst Neurosci, CH-1015 Lausanne, Switzerland Ecole Polytech Fed Lausanne, Swiss Fed Inst Technol, Inst Neurosci, CH-1015 Lausanne, Switzerland

Déglon, N
论文数: 0 引用数: 0
h-index: 0
机构:
Ecole Polytech Fed Lausanne, Swiss Fed Inst Technol, Inst Neurosci, CH-1015 Lausanne, Switzerland Ecole Polytech Fed Lausanne, Swiss Fed Inst Technol, Inst Neurosci, CH-1015 Lausanne, Switzerland

Pralong, W
论文数: 0 引用数: 0
h-index: 0
机构:
Ecole Polytech Fed Lausanne, Swiss Fed Inst Technol, Inst Neurosci, CH-1015 Lausanne, Switzerland Ecole Polytech Fed Lausanne, Swiss Fed Inst Technol, Inst Neurosci, CH-1015 Lausanne, Switzerland

Aebischer, P
论文数: 0 引用数: 0
h-index: 0
机构:
Ecole Polytech Fed Lausanne, Swiss Fed Inst Technol, Inst Neurosci, CH-1015 Lausanne, Switzerland Ecole Polytech Fed Lausanne, Swiss Fed Inst Technol, Inst Neurosci, CH-1015 Lausanne, Switzerland
[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

Aitken, ML
论文数: 0 引用数: 0
h-index: 0
机构: Univ Washington, Dept Med & Pediat, Seattle, WA 98195 USA

Moss, RB
论文数: 0 引用数: 0
h-index: 0
机构: Univ Washington, Dept Med & Pediat, Seattle, WA 98195 USA

Waltz, DA
论文数: 0 引用数: 0
h-index: 0
机构: Univ Washington, Dept Med & Pediat, Seattle, WA 98195 USA

Dovey, ME
论文数: 0 引用数: 0
h-index: 0
机构: Univ Washington, Dept Med & Pediat, Seattle, WA 98195 USA

Tonelli, MR
论文数: 0 引用数: 0
h-index: 0
机构: Univ Washington, Dept Med & Pediat, Seattle, WA 98195 USA

McNamara, SC
论文数: 0 引用数: 0
h-index: 0
机构: Univ Washington, Dept Med & Pediat, Seattle, WA 98195 USA

Gibson, RL
论文数: 0 引用数: 0
h-index: 0
机构: Univ Washington, Dept Med & Pediat, Seattle, WA 98195 USA

Ramsey, BW
论文数: 0 引用数: 0
h-index: 0
机构: Univ Washington, Dept Med & Pediat, Seattle, WA 98195 USA

Carter, BJ
论文数: 0 引用数: 0
h-index: 0
机构: Univ Washington, Dept Med & Pediat, Seattle, WA 98195 USA

Reynolds, TC
论文数: 0 引用数: 0
h-index: 0
机构: Univ Washington, Dept Med & Pediat, Seattle, WA 98195 USA
[4]
Biomedicine - Lentiviral vectors - the promise of gene therapy within reach?
[J].
Amado, RG
;
Chen, ISY
.
SCIENCE,
1999, 285 (5428)
:674-676

Amado, RG
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Calif Los Angeles, Sch Med, Dept Microbiol & Immunol, Div Hematol Oncol, Los Angeles, CA 90095 USA Univ Calif Los Angeles, Sch Med, Dept Microbiol & Immunol, Div Hematol Oncol, Los Angeles, CA 90095 USA

Chen, ISY
论文数: 0 引用数: 0
h-index: 0
机构: Univ Calif Los Angeles, Sch Med, Dept Microbiol & Immunol, Div Hematol Oncol, Los Angeles, CA 90095 USA
[5]
PROSPECTS FOR HUMAN-GENE THERAPY
[J].
ANDERSON, WF
.
SCIENCE,
1984, 226 (4673)
:401-409

ANDERSON, WF
论文数: 0 引用数: 0
h-index: 0
[6]
HUMAN GENE-THERAPY
[J].
ANDERSON, WF
.
SCIENCE,
1992, 256 (5058)
:808-813

ANDERSON, WF
论文数: 0 引用数: 0
h-index: 0
[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

ANDERSON, WF
论文数: 0 引用数: 0
h-index: 0

FLETCHER, JC
论文数: 0 引用数: 0
h-index: 0
[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

Andreoletti, M
论文数: 0 引用数: 0
h-index: 0
机构: Hop Antoine Beclere, INSERM, EMI 0020, F-92141 Clamart, France

Loux, N
论文数: 0 引用数: 0
h-index: 0
机构: Hop Antoine Beclere, INSERM, EMI 0020, F-92141 Clamart, France

Vons, C
论文数: 0 引用数: 0
h-index: 0
机构: Hop Antoine Beclere, INSERM, EMI 0020, F-92141 Clamart, France

Nguyen, TH
论文数: 0 引用数: 0
h-index: 0
机构: Hop Antoine Beclere, INSERM, EMI 0020, F-92141 Clamart, France

Lorand, I
论文数: 0 引用数: 0
h-index: 0
机构: Hop Antoine Beclere, INSERM, EMI 0020, F-92141 Clamart, France

Mahieu, D
论文数: 0 引用数: 0
h-index: 0
机构: Hop Antoine Beclere, INSERM, EMI 0020, F-92141 Clamart, France

Simon, L
论文数: 0 引用数: 0
h-index: 0
机构: Hop Antoine Beclere, INSERM, EMI 0020, F-92141 Clamart, France

Di Rico, V
论文数: 0 引用数: 0
h-index: 0
机构: Hop Antoine Beclere, INSERM, EMI 0020, F-92141 Clamart, France

Vingert, B
论文数: 0 引用数: 0
h-index: 0
机构: Hop Antoine Beclere, INSERM, EMI 0020, F-92141 Clamart, France

Chapman, J
论文数: 0 引用数: 0
h-index: 0
机构: Hop Antoine Beclere, INSERM, EMI 0020, F-92141 Clamart, France

Briand, P
论文数: 0 引用数: 0
h-index: 0
机构: Hop Antoine Beclere, INSERM, EMI 0020, F-92141 Clamart, France

Schwall, R
论文数: 0 引用数: 0
h-index: 0
机构: Hop Antoine Beclere, INSERM, EMI 0020, F-92141 Clamart, France

Hamza, J
论文数: 0 引用数: 0
h-index: 0
机构: Hop Antoine Beclere, INSERM, EMI 0020, F-92141 Clamart, France

Capron, F
论文数: 0 引用数: 0
h-index: 0
机构: Hop Antoine Beclere, INSERM, EMI 0020, F-92141 Clamart, France

Bargy, F
论文数: 0 引用数: 0
h-index: 0
机构: Hop Antoine Beclere, INSERM, EMI 0020, F-92141 Clamart, France

Franco, D
论文数: 0 引用数: 0
h-index: 0
机构: Hop Antoine Beclere, INSERM, EMI 0020, F-92141 Clamart, France

Weber, A
论文数: 0 引用数: 0
h-index: 0
机构:
Hop Antoine Beclere, INSERM, EMI 0020, F-92141 Clamart, France Hop Antoine Beclere, INSERM, EMI 0020, F-92141 Clamart, France
[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

BANAI, S
论文数: 0 引用数: 0
h-index: 0
机构:
NHLBI,CARDIOL BRANCH,BETHESDA,MD 20892 NHLBI,CARDIOL BRANCH,BETHESDA,MD 20892

JAKLITSCH, MT
论文数: 0 引用数: 0
h-index: 0
机构:
NHLBI,CARDIOL BRANCH,BETHESDA,MD 20892 NHLBI,CARDIOL BRANCH,BETHESDA,MD 20892

SHOU, M
论文数: 0 引用数: 0
h-index: 0
机构:
NHLBI,CARDIOL BRANCH,BETHESDA,MD 20892 NHLBI,CARDIOL BRANCH,BETHESDA,MD 20892

LAZAROUS, DF
论文数: 0 引用数: 0
h-index: 0
机构:
NHLBI,CARDIOL BRANCH,BETHESDA,MD 20892 NHLBI,CARDIOL BRANCH,BETHESDA,MD 20892

SCHEINOWITZ, M
论文数: 0 引用数: 0
h-index: 0
机构:
NHLBI,CARDIOL BRANCH,BETHESDA,MD 20892 NHLBI,CARDIOL BRANCH,BETHESDA,MD 20892

BIRO, S
论文数: 0 引用数: 0
h-index: 0
机构:
NHLBI,CARDIOL BRANCH,BETHESDA,MD 20892 NHLBI,CARDIOL BRANCH,BETHESDA,MD 20892

EPSTEIN, SE
论文数: 0 引用数: 0
h-index: 0
机构:
NHLBI,CARDIOL BRANCH,BETHESDA,MD 20892 NHLBI,CARDIOL BRANCH,BETHESDA,MD 20892

UNGER, EF
论文数: 0 引用数: 0
h-index: 0
机构:
NHLBI,CARDIOL BRANCH,BETHESDA,MD 20892 NHLBI,CARDIOL BRANCH,BETHESDA,MD 20892
[10]
RNA interference - The short answer
[J].
Bass, BL
.
NATURE,
2001, 411 (6836)
:428-429

Bass, BL
论文数: 0 引用数: 0
h-index: 0
机构: Univ Utah, Dept Biochem, Salt Lake City, UT 84132 USA