Retroviral integration and human gene therapy

被引：95

作者：

Bushman, Frederic D. ^{[1
]}

机构：

[1] Univ Penn, Sch Med, Dept Microbiol, Philadelphia, PA 19104 USA

来源：

JOURNAL OF CLINICAL INVESTIGATION | 2007年 / 117卷 / 08期

关键词：

D O I：

10.1172/JCI32949

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

Long-term correction of genetic diseases requires permanent integration of therapeutic genes into chromosomes of affected cells. Retroviral vectors are the most widely used delivery vehicles because of their efficiency and precision of integration. However, retroviral integration can take place at a variety of chromosomal sites, and examples have been reported of integration of therapeutic vectors activating oncogenes and causing cancer in patients. This issue of the JCI presents three articles that update successful human gene therapy trials and furthermore evaluate the sites of integration in cells from treated patients, including samples from individuals experiencing serious adverse events following therapy (see the related articles beginning on pages 2225, 2233, and 2241).

引用

页码：2083 / 2086

页数：4

共 25 条

[1] Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning [J].

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 .

SCIENCE, 2002, 296 (5577) :2410-2413

[2] Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy [J].

Aiuti, Alessandro ;

Cassani, Barbara ;

Andolfi, Grazia ;

Mirolo, Massimiliano ;

Biasco, Luca ;

Recchia, Alessandra ;

Urbinati, Fabrizia ;

Valacca, Cristina ;

Scaramuzza, Samantha ;

Aker, Memet ;

Slavin, Shimon ;

Cazzola, Matteo ;

Sartori, Daniela ;

Ambrosi, Alessandro ;

Di Serio, Clelia ;

Roncarolo, Maria Grazia ;

Mavilio, Fulvio ;

Bordignon, Claudio .

JOURNAL OF CLINICAL INVESTIGATION, 2007, 117 (08) :2233-2240

[3] Selection of target sites for mobile DNA integration in the human genome [J].

Berry, Charles ;

Hannenhalli, Sridhar ;

Leipzig, Jeremy ;

Bushman, Frederic D. .

PLOS COMPUTATIONAL BIOLOGY, 2006, 2 (11) :1450-1462

[4] 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

[5] Genome wide analysis of retroviral DNA integration [J].

Bushman, F ;

Lewinski, M ;

Ciuffi, A ;

Barr, S ;

Leipzig, J ;

Hannenhalli, S ;

Hoffmann, C .

NATURE REVIEWS MICROBIOLOGY, 2005, 3 (11) :848-858

[6]

Bushman F.D., 2001, LATERAL DNA TRANSFER

[7] Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease [J].

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 .

SCIENCE, 2000, 288 (5466) :669-672

[8]

Craig N., 2002, MOBILE DNA

[9] Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy [J].

Deichmann, Annette ;

Hacein-Bey-Abina, Salima ;

Schmidt, Manfred ;

Garrigue, Alexandrine ;

Brugman, Martijn H. ;

Hu, Jingqiong ;

Glimm, Hanno ;

Gyapay, Gabor ;

Prum, Bernard ;

Fraser, Christopher C. ;

Fischer, Nicolas ;

Schwarzwaelder, Kerstin ;

Siegler, Maria-Luise ;

de Ridder, Dick ;

Pike-Overzet, Karin ;

Howe, Steven J. ;

Thrasher, Adrian J. ;

Wagemaker, Gerard ;

Abel, Ulrich ;

Staal, Frank J. T. ;

Delabesse, Eric ;

Villeval, Jean-Luc ;

Aronow, Bruce ;

Hue, Christophe ;

Prinz, Claudia ;

Wissler, Manuela ;

Klanke, Chuck ;

Weissenbach, Jean ;

Alexander, Ian ;

Fischer, Alain ;

von Kalle, Christof ;

Cavazzana-Calvo, Marina .

JOURNAL OF CLINICAL INVESTIGATION, 2007, 117 (08) :2225-2232

[10] LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 [J].

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 .

SCIENCE, 2003, 302 (5644) :415-419

← 1 2 3 →