Integrating Vectors for Gene Therapy and Clonal Tracking of Engineered Hematopoiesis

被引：13

作者：

Biasco, Luca ^{[1
,2
]}

Rothe, Michael ^{[3
]}

Schott, Juliane W. ^{[3
]}

Schambach, Axel ^{[3
,4
]}

机构：

[1] Harvard Med Sch, Dana Farber Boston Childrens Canc & Blood Disorde, Gene Therapy Program, 1 Jimmy Fund Way, Boston, MA 02115 USA

[2] UCL, UCL Great Ormond St Inst Child Hlth, UCL Fac Populat Hlth Sci, 30 Guilford St, London WC1N 1EH, England

[3] Hannover Med Sch, REBIRTH Cluster Excellence, Inst Expt Hematol, Carl Neuberg Str 1, D-30625 Hannover, Germany

[4] Harvard Med Sch, Div Hematol Oncol, Boston Childrens Hosp, 300 Longwood Ave, Boston, MA 02115 USA

来源：

HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA | 2017年 / 31卷 / 05期

关键词：

Retroviral vector; Lentiviral vector; Transposon; Stem cells; Gene therapy; Clonal tracking; Insertion site analysis; Hematopoietic dynamics; LEUKEMIA-VIRUS INTEGRATION; HSV-TK GENE; LENTIVIRAL VECTOR; INSERTIONAL MUTAGENESIS; SLEEPING-BEAUTY; RETROVIRAL INTEGRATION; HIV-1; INTEGRATION; HUMAN GENOME; CELLS; SCID-X1;

D O I：

10.1016/j.hoc.2017.06.009

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Gene therapy using autologous or allogeneic cells offers promising possibilities to treat inherited and acquired diseases, ideally leading to a long lasting therapeutic correction. This article summarizes efforts that use integrating vectors derived from retroviruses and transposons, and briefly explains integrating vector biology and integration site analysis and recent successful application of this technology in clinical trials. Moreover, the article outlines how these vectors can be used for cancer gene discovery and clonal tracking of benign and malignant hematopoiesis to gain insights into the dynamics of hematopoiesis.

引用

页码：737 / +

页数：17

共 94 条

[91] Lentiviral Vector Design and Imaging Approaches to Visualize the Early Stages of Cellular Reprogramming [J].

Warlich, Eva ;

Kuehle, Johannes ;

Cantz, Tobias ;

Brugman, Martijn H. ;

Maetzig, Tobias ;

Galla, Melanie ;

Filipczyk, Adam A. ;

Halle, Stephan ;

Klump, Hannes ;

Schoeler, Hans R. ;

Baum, Christopher ;

Schroeder, Timm ;

Schambach, Axel .

MOLECULAR THERAPY, 2011, 19 (04) :782-789

[92] Transcription start regions in the human genome are favored targets for MLV integration [J].

Wu, XL ;

Li, Y ;

Crise, B ;

Burgess, SM .

SCIENCE, 2003, 300 (5626) :1749-1751

[93] SELF-INACTIVATING RETROVIRAL VECTORS DESIGNED FOR TRANSFER OF WHOLE GENES INTO MAMMALIAN-CELLS [J].

YU, SF ;

VONRUDEN, T ;

KANTOFF, PW ;

GARBER, C ;

SEIBERG, M ;

RUTHER, U ;

ANDERSON, WF ;

WAGNER, EF ;

GILBOA, E .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1986, 83 (10) :3194-3198

[94] A hyperactive piggyBac transposase for mammalian applications [J].

Yusa, Kosuke ;

Zhou, Liqin ;

Li, Meng Amy ;

Bradley, Allan ;

Craig, Nancy L. .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2011, 108 (04) :1531-1536

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