A new chance for SCID gene therapy

被引:10
作者
Bordignon, C [1 ]
机构
[1] San Raffaele Sci Inst, I-20132 Milan, Italy
关键词
D O I
10.1038/nm0198-019
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
Constitutive expression of a Jak-3 transgene reverses severe combined immunodeficiency in a mouse model (pages 58–64).
引用
收藏
页码:19 / 20
页数:2
相关论文
共 11 条
[1]   PROSPECTS FOR HUMAN-GENE THERAPY [J].
ANDERSON, WF .
SCIENCE, 1984, 226 (4673) :401-409
[2]   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
[3]   GENE-THERAPY IN PERIPHERAL-BLOOD LYMPHOCYTES AND BONE-MARROW FOR ADA(-) IMMUNODEFICIENT PATIENTS [J].
BORDIGNON, C ;
NOTARANGELO, LD ;
NOBILI, N ;
FERRARI, G ;
CASORATI, G ;
PANINA, P ;
MAZZOLARI, E ;
MAGGIONI, D ;
ROSSI, C ;
SERVIDA, P ;
UGAZIO, AG ;
MAVILIO, F .
SCIENCE, 1995, 270 (5235) :470-475
[4]   Restoration of lymphocyte function in Janus kinase 3-deficient mice by retroviral-mediated gene transfer [J].
Bunting, KD ;
Sangster, MY ;
Ihle, JN ;
Sorrentino, BP .
NATURE MEDICINE, 1998, 4 (01) :58-64
[5]   In vitro correction of JAK3-deficient severe combined immunodeficiency by retroviral-mediated gene transduction [J].
Candotti, F ;
Oakes, SA ;
Johnston, JA ;
Notarangelo, LD ;
OShea, JJ ;
Blaese, RM .
JOURNAL OF EXPERIMENTAL MEDICINE, 1996, 183 (06) :2687-2692
[6]  
CANDOTTI F, 1997, BLOOD, V10, P3995
[7]   AN INVIVO MODEL OF SOMATIC-CELL GENE-THERAPY FOR HUMAN SEVERE COMBINED IMMUNODEFICIENCY [J].
FERRARI, G ;
ROSSINI, S ;
GIAVAZZI, R ;
MAGGIONI, D ;
NOBILI, N ;
SOLDATI, M ;
UNGERS, G ;
MAVILIO, F ;
GILBOA, E ;
BORDIGNON, C .
SCIENCE, 1991, 251 (4999) :1363-1366
[8]  
Hoogerbrugge PM, 1996, GENE THER, V3, P179
[9]   ENGRAFTMENT OF GENE-MODIFIED UMBILICAL-CORD BLOOD-CELLS IN NEONATES WITH ADENOSINE-DEAMINASE DEFICIENCY [J].
KOHN, DB ;
WEINBERG, KI ;
NOLTA, JA ;
HEISS, LN ;
LENARSKY, C ;
CROOKS, GM ;
HANLEY, ME ;
ANNETT, G ;
BROOKS, JS ;
ELKHOUREIY, A ;
LAWRENCE, K ;
WELLS, S ;
MOEN, RC ;
BASTIAN, J ;
WILLIAMSHERMAN, DE ;
ELDER, M ;
WARA, D ;
BOWEN, T ;
HERSHFIELD, MS ;
MULLEN, CA ;
BLAESE, RM ;
PARKMAN, R .
NATURE MEDICINE, 1995, 1 (10) :1017-1023
[10]   MUTATIONS OF JAK-3 GENE IN PATIENTS WITH AUTOSOMAL SEVERE COMBINED IMMUNE-DEFICIENCY (SCID) [J].
MACCHI, P ;
VILLA, A ;
GILIANI, S ;
SACCO, MG ;
FRATTINI, A ;
PORTA, F ;
UGAZIO, AG ;
JOHNSTON, JA ;
CANDOTTI, F ;
O'SHEA, JJ ;
VEZZONI, P ;
NOTARANGELO, LD .
NATURE, 1995, 377 (6544) :65-68