Gene therapy: lessons learnt from the past decade

被引:10
作者
Morgan, RA
Blaese, RM
机构
[1] NIH, Clin Gene Therapy Branch, Bethesda, MD 20892 USA
[2] Kimeragen Inc, Newtown, PA 18940 USA
关键词
D O I
10.1136/bmj.319.7220.1310
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
If is more than a decade since the first approved clinical trial to put genes into the tells of human beings was initiated, In that first trial, investigators at the US National Institutes of Health used a modified mouse leukaemia virus to insert a DNA marker into lymphocytes being used to treat cancer(1). A year later, a similar viral technique was used as treatment for two gills who had severe combined immunodeficiency with mutant adenosine deaminase,(2) Ten years and more than 3000 patients later, it is appropriate to ask, "Where are we now?"
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页码:1310 / U85
页数:3
相关论文
共 14 条
[1]   T-LYMPHOCYTE-DIRECTED GENE-THERAPY FOR ADA(-) SCID - INITIAL TRIAL RESULTS AFTER 4 YEARS [J].
BLAESE, RM ;
CULVER, KW ;
MILLER, AD ;
CARTER, CS ;
FLEISHER, T ;
CLERICI, M ;
SHEARER, G ;
CHANG, L ;
CHIANG, YW ;
TOLSTOSHEV, P ;
GREENBLATT, JJ ;
ROSENBERG, SA ;
KLEIN, H ;
BERGER, M ;
MULLEN, CA ;
RAMSEY, WJ ;
MUUL, L ;
MORGAN, RA ;
ANDERSON, WF .
SCIENCE, 1995, 270 (5235) :475-480
[2]   Correction of the mutation responsible for sickle cell anemia by an RNA-DNA oligonucleotide [J].
ColeStrauss, A ;
Yoon, KG ;
Xiang, YF ;
Byrne, BC ;
Rice, MC ;
Gryn, J ;
Holloman, WK ;
Kmiec, EB .
SCIENCE, 1996, 273 (5280) :1386-1389
[3]   DNA vaccines [J].
Donnelly, JJ ;
Ulmer, JB ;
Shiver, JW ;
Liu, MA .
ANNUAL REVIEW OF IMMUNOLOGY, 1997, 15 :617-648
[4]   RETROVIRALLY MARKED CD34-ENRICHED PERIPHERAL-BLOOD AND BONE-MARROW CELLS CONTRIBUTE TO LONG-TERM ENGRAFTMENT AFTER AUTOLOGOUS TRANSPLANTATION [J].
DUNBAR, CE ;
COTTLERFOX, M ;
OSHAUGHNESSY, JA ;
DOREN, S ;
CARTER, C ;
BERENSON, R ;
BROWN, S ;
MOEN, RC ;
GREENBLATT, J ;
STEWART, FM ;
LEITMAN, SF ;
WILSON, WH ;
COWAN, K ;
YOUNG, NS ;
NIENHUIS, AW .
BLOOD, 1995, 85 (11) :3048-3057
[5]   Nucleic acid therapeutics: State of the art and future prospects [J].
Gewirtz, AM ;
Sokol, DL ;
Ratajczak, MZ .
BLOOD, 1998, 92 (03) :712-736
[6]   Science, medicine, and the future - Therapeutic angiogenesis [J].
Henry, TD .
BMJ-BRITISH MEDICAL JOURNAL, 1999, 318 (7197) :1536-1539
[7]   In vivo site-directed mutagenesis of the factor IX gene by chimeric RNA/DNA oligonucleotides [J].
Kren, BT ;
Bandyopadhyay, P ;
Steer, CJ .
NATURE MEDICINE, 1998, 4 (03) :285-290
[8]   The kinetics of rAAV integration in the liver [J].
Miao, CH ;
Snyder, RO ;
Schowalter, DB ;
Patijn, GA ;
Donahue, B ;
Winther, B ;
Kay, MA .
NATURE GENETICS, 1998, 19 (01) :13-15
[9]  
MUZYCZKA N, 1992, CURR TOP MICROBIOL, V158, P97
[10]   T-cell mediated rejection of gene-modified HIV-specific cytotoxic T lymphocytes in HIV-infected patients [J].
Riddell, SR ;
Elliott, M ;
Lewinsohn, DA ;
Gilbert, MJ ;
Wilson, L ;
Manley, SA ;
Lupton, SD ;
Overell, RW ;
Reynolds, TC ;
Corey, L ;
Greenberg, PD .
NATURE MEDICINE, 1996, 2 (02) :216-223