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.
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收藏
页码：439 / 443
页数：5
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