The future of gene therapy

被引：186

作者：

Cavazzana-Calvo, M ^{[1
]}

Thrasher, A

Mavilio, F

机构：

[1] Hop Necker Enfants Malad, Immunol & Pediat Haematol Unit, F-75743 Paris 15, France

[2] Inst Child Hlth, London WC1N 1EH, England

[3] Ist Sci H San Raffaele, I-20132 Milan, Italy

[4] Univ Modena, Dept Biomed Sci, I-41100 Modena, Italy

来源：

NATURE | 2004年 / 427卷 / 6977期

关键词：

D O I：

10.1038/427779a

中图分类号：

O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

Balancing the risks and benefits of clinical trial.

引用

页码：779 / 781

页数：3

共 9 条

[1] Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning
Aiuti, A
Slavin, S
Aker, M
Ficara, F
Deola, S
Mortellaro, A
Morecki, S
Andolfi, G
Tabucchi, A
Carlucci, F
Marinello, E
Cattaneo, F
Vai, S
Servida, P
Miniero, R
Roncarolo, MG
Bordignon, C
[J]. SCIENCE, 2002, 296 (5577) : 2410 - 2413
[2] Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies:: report of the European experience 1968-99
Antoine, C
Müller, S
Cant, A
Cavazzana-Calvo, M
Veys, P
Vossen, J
Fasth, A
Heilmann, C
Wulffraat, N
Seger, R
Blanche, S
Friedrich, W
Abinun, M
Davies, G
Bredius, R
Schulz, A
Landais, P
Fischer, A
[J]. LANCET, 2003, 361 (9357) : 553 - 560
[3] Side effects of retroviral gene transfer into hematopoietic stem cells
Baum, C
Düllmann, J
Li, ZX
Fehse, B
Meyer, J
Williams, DA
von Kalle, C
[J]. BLOOD, 2003, 101 (06) : 2099 - 2114
[4] Safety of retroviral gene marking with a truncated NGF receptor
Bonini, C
Grez, M
Traversari, C
Ciceri, F
Marktel, S
Ferrari, G
Dinauer, M
Sadat, M
Aiuti, A
Deola, S
Radrizzani, M
Hagenbeek, A
Apperley, J
Ebeling, S
Martens, A
Kolb, HJ
Weber, M
Lotti, F
Grande, A
Weissinger, E
Bueren, JA
Lamana, M
Falkenburg, JHF
Heemskerk, MHM
Austin, T
Kornblau, S
Marini, F
Benati, C
Magnani, Z
Cazzaniga, S
Toma, S
Gallo-Stampino, C
Introna, M
Slavin, S
Greenberg, PD
Bregni, M
Mavilio, F
Bordignon, C
[J]. NATURE MEDICINE, 2003, 9 (04) : 367 - 369
[5] Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
Cavazzana-Calvo, M
Hacein-Bey, S
Basile, CD
Gross, F
Yvon, E
Nusbaum, P
Selz, F
Hue, C
Certain, S
Casanova, JL
Bousso, P
Le Deist, F
Fischer, A
[J]. SCIENCE, 2000, 288 (5466) : 669 - 672
[6] Gene therapy insertional mutagenesis insights
Davé, UP
Jenkins, NA
Copeland, NG
[J]. SCIENCE, 2004, 303 (5656) : 333 - 333
[7] Primary immunodeficiency diseases: an experimental model for molecular medicine
Fischer, A
[J]. LANCET, 2001, 357 (9271) : 1863 - 1869
[8] LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
Hacein-Bey-Abina, S
Von Kalle, C
Schmidt, M
McCcormack, MP
Wulffraat, N
Leboulch, P
Lim, A
Osborne, CS
Pawliuk, R
Morillon, E
Sorensen, R
Forster, A
Fraser, P
Cohen, JI
de Saint Basile, G
Alexander, I
Wintergerst, U
Frebourg, T
Aurias, A
Stoppa-Lyonnet, D
Romana, S
Radford-Weiss, I
Gross, F
Valensi, F
Delabesse, E
Macintyre, E
Sigaux, F
Soulier, J
Leiva, LE
Wissler, M
Prinz, C
Rabbitts, TH
Le Deist, F
Fischer, A
Cavazzana-Calvo, M
[J]. SCIENCE, 2003, 302 (5644) : 415 - 419
[9] Gene therapy - New challenges ahead
Williams, DA
Baum, C
[J]. SCIENCE, 2003, 302 (5644) : 400 - 401

← 1 →