EXPRESSION OF HUMAN ADENOSINE-DEAMINASE IN MICE RECONSTITUTED WITH RETROVIRUS-TRANSDUCED HEMATOPOIETIC STEM-CELLS

被引：114

作者：

WILSON, JM

DANOS, O

GROSSMAN, M

RAULET, DH

MULLIGAN, RC

机构：

[1] MIT,WHITEHEAD INST BIOMED RES,CAMBRIDGE,MA 02142

[2] MIT,DEPT BIOL,CAMBRIDGE,MA 02142

[3] MIT,CTR CANC RES,CAMBRIDGE,MA 02142

来源：

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA | 1990年 / 87卷 / 01期

关键词：

Bone marrow transplantation; Gene therapy; Retroviral vector;

D O I：

10.1073/pnas.87.1.439

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：

Recombinant retroviruses encoding human adenosine deaminase (ADA; adenosine aminohydrolase, EC 3.5.4.4) have been used to infect murine hematopoietic stem cells. In bone marrow transplant recipients reconstituted with the genetically modified cells, human ADA was detected in peripheral blood mononuclear cells of the recipients for at least 6 months after transplantation. In animals analyzed in detail 4 months after transplantation, human ADA and proviral sequences were detected in all hematopoietic lineages; in several cases, human ADA activity exceeded the endogenous activity. These studies demonstrate the feasibility of introducing a functional human ADA gene into hematopoietic stem cells and obtaining expression in multiple hematopoietic lineages long after transplantation. This approach should be helpful in designing effective gene therapies for severe combined immunodeficiency syndromes in humans.

引用

页码：439 / 443

页数：5

共 50 条

[41] RESTRICTION OF EXPRESSION OF AN INTEGRATED RECOMBINANT RETROVIRUS IN PRIMARY BUT NOT IMMORTALIZED MURINE HEMATOPOIETIC STEM-CELLS
WILLIAMS, DA
LIM, B
SPOONCER, E
LONGTINE, J
DEXTER, TM
BLOOD, 1988, 71 (06) : 1738 - 1743
[42] PARADOXICAL EXPRESSION OF ADENOSINE-DEAMINASE IN T-CELLS CULTURED FROM A PATIENT WITH ADENOSINE-DEAMINASE DEFICIENCY AND COMBINED IMMUNODEFICIENCY
ARREDONDOVEGA, FX
KURTZBERG, J
CHAFFEE, S
SANTISTEBAN, I
REISNER, E
POVEY, MS
HERSHFIELD, MS
JOURNAL OF CLINICAL INVESTIGATION, 1990, 86 (02): : 444 - 452
[43] RETROVIRAL-MEDIATED TRANSFER OF HUMAN ADENOSINE-DEAMINASE IN MICE
OSBORNE, WRA
KALEKO, M
GARCIA, JV
MILLER, AD
CLINICAL RESEARCH, 1990, 38 (02): : A387 - A387
[44] EXPRESSION OF HUMAN ADENOSINE-DEAMINASE IN NONHUMAN-PRIMATES AFTER RETROVIRUS-MEDIATED GENE-TRANSFER
KANTOFF, PW
GILLIO, AP
MCLACHLIN, JR
BORDIGNON, C
EGLITIS, MA
KERNAN, NA
MOEN, RC
KOHN, DB
YU, SF
KARSON, E
KARLSSON, S
ZWIEBEL, JA
GILBOA, E
BLAESE, RM
NIENHUIS, A
OREILLY, RJ
ANDERSON, WF
JOURNAL OF EXPERIMENTAL MEDICINE, 1987, 166 (01): : 219 - 234
[45] EXPRESSION OF HUMAN GLUCOCEREBROSIDASE IN LONG-TERM RECONSTITUTED MICE FOLLOWING RETROVIRAL-MEDIATED GENE-TRANSFER INTO HEMATOPOIETIC STEM-CELLS
CORRELL, PH
KEW, Y
PERRY, LK
BRADY, RO
FINK, JK
KARLSSON, S
HUMAN GENE THERAPY, 1990, 1 (03) : 277 - 287
[46] EXPRESSION VECTORS FOR HUMAN ADENOSINE-DEAMINASE GENE-THERAPY
MOORE, KA
FLETCHER, FA
ALFORD, RL
VILLALON, DK
HAWKINS, DH
MACGREGOR, GR
CASKEY, CT
BELMONT, JW
GENOME, 1989, 31 (02) : 832 - 839
[47] A MAJORITY OF MICE SHOW LONG-TERM EXPRESSION OF A HUMAN BETA-GLOBIN GENE AFTER RETROVIRUS TRANSFER INTO HEMATOPOIETIC STEM-CELLS
BENDER, MA
GELINAS, RE
MILLER, AD
MOLECULAR AND CELLULAR BIOLOGY, 1989, 9 (04) : 1426 - 1434
[48] MUTANT HUMAN ADENOSINE-DEAMINASE ALLELES AND THEIR EXPRESSION BY TRANSFECTION INTO FIBROBLASTS
AKESON, AL
WIGINTON, DA
DUSING, MR
STATES, JC
HUTTON, JJ
JOURNAL OF BIOLOGICAL CHEMISTRY, 1988, 263 (31) : 16291 - 16296
[49] GENETIC EXPRESSION OF ADENOSINE-DEAMINASE (ADA) IN HUMAN LYMPHOID MALIGNANCIES
GAN, TE
DADDONA, P
MITCHELL, B
PEDIATRIC RESEARCH, 1985, 19 (07) : 755 - 755
[50] THE HUMAN-GENE FOR ADENOSINE-DEAMINASE - ITS STRUCTURAL ORGANIZATION AND EXPRESSION IN MOUSE CELLS
VALERIO, D
BERKVENS, TM
DUYVESTEYN, MGC
VANDERVOORN, L
DEKKER, BMM
VANORMONDT, H
KHAN, PM
VANDEREB, AJ
ANNALS OF THE NEW YORK ACADEMY OF SCIENCES, 1985, 451 (OCT) : 332 - 335

← 1 2 3 4 5 →