Gene therapy of hematopoietic stem cells: Strategies for improvement

被引：8

作者：

Hossle, JP ^{[1
]}

Seger, RA ^{[1
]}

Steinhoff, D ^{[1
]}

机构：

[1] Univ Zurich, Childrens Hosp, Div Immunol Hematol Oncol, CH-8032 Zurich, Switzerland

来源：

NEWS IN PHYSIOLOGICAL SCIENCES | 2002年 / 17卷

关键词：

D O I：

10.1152/nips.01343.2001

中图分类号：

Q4 [生理学];

学科分类号：

071003 ;

摘要：

Gene therapy of hematopoietic stem cells (HSC) is limited by low frequency of the target cells, their quiescent nature, poor engraftment of treated HSC, and lack of a selective growth advantage of genetically modified cells. Lentiviral vectors combined with positive selection strategies using conditional cell-growth switches should allow for improvement.

引用

页码：87 / 92

页数：6

共 21 条

[1] MYELOSUPPRESSIVE CONDITIONING IMPROVES AUTOLOGOUS ENGRAFTMENT OF GENETICALLY MARKED HEMATOPOIETIC REPOPULATING CELLS IN DOGS [J].

BARQUINERO, J ;

KIEM, HP ;

VONKALLE, C ;

DAROVSKY, B ;

GOEHLE, S ;

GRAHAM, T ;

SEIDEL, K ;

STORB, R ;

SCHUENING, FG .

BLOOD, 1995, 85 (05) :1195-1201

[2] Correction of respiratory burst activity in X-linked chronic granulomatous cells to therapeutically relevant levels after gene transfer into bone marrow CD34+ cells [J].

Becker, S ;

Wasser, S ;

Hauses, M ;

Hossle, JP ;

Ott, MG ;

Dinauer, MC ;

Ganser, A ;

Hoelzer, D ;

Seger, R ;

Grez, M .

HUMAN GENE THERAPY, 1998, 9 (11) :1561-1570

[3]

Brenner S, 2000, BLOOD, V96, p430A

[4] Transduction of murine bone marrow cells with an MDR1 vector enables ex vivo stem cell expansion, but these expanded grafts cause a myeloproliferative syndrome in transplanted mice [J].

Bunting, KD ;

Galipeau, J ;

Topham, D ;

Benaim, E ;

Sorrentino, BP .

BLOOD, 1998, 92 (07) :2269-2279

[5] 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

[6]

Choi UM, 2000, BLOOD, V96, p805A

[7] Human hematopoietic stem cells stimulated to proliferate in vitro lose engraftment potential during their S/G2/M transit and do not reenter G0 [J].

Glimm, H ;

Oh, IL ;

Eaves, CJ .

BLOOD, 2000, 96 (13) :4185-4193

[8] Direct evidence for multiple self-renewal divisions of human in vivo repopulating hematopoietic cells in short-term culture [J].

Glimm, H ;

Eaves, CJ .

BLOOD, 1999, 94 (07) :2161-2168

[9] The fluctuating phenotype of the lymphohematopoietic stem cell with cell cycle transit [J].

Habibian, HK ;

Peters, SO ;

Hsieh, CC ;

Wuu, J ;

Vergilis, K ;

Grimaldi, CI ;

Reilly, J ;

Carlson, JE ;

Frimberger, AE ;

Stewart, FM ;

Quesenberry, PJ .

JOURNAL OF EXPERIMENTAL MEDICINE, 1998, 188 (02) :393-398

[10] Targeted expansion of genetically modified bone marrow cells [J].

Jin, LQ ;

Siritanaratkul, N ;

Emery, DW ;

Richard, RE ;

Kaushansky, K ;

Papayannopoulou, T ;

Blau, CA .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1998, 95 (14) :8093-8097

← 1 2 3 →