Wilson J M, Danos O, Grossman M, Raulet D H, Mulligan R C
Whitehead Institute for Biomedical Research, Cambridge, MA.
Proc Natl Acad Sci U S A. 1990 Jan;87(1):439-43. doi: 10.1073/pnas.87.1.439.
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.
编码人腺苷脱氨酶（ADA；腺苷氨基水解酶，EC 3.5.4.4）的重组逆转录病毒已被用于感染小鼠造血干细胞。在用基因改造细胞重建后的骨髓移植受者中，移植后至少6个月在受者外周血单核细胞中检测到了人ADA。在移植后4个月进行详细分析的动物中，在所有造血谱系中均检测到了人ADA和前病毒序列；在一些情况下，人ADA活性超过了内源性活性。这些研究证明了将功能性人ADA基因导入造血干细胞并在移植后很长时间内在多个造血谱系中获得表达的可行性。这种方法应有助于设计针对人类严重联合免疫缺陷综合征的有效基因疗法。
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