基因修饰的脐带血细胞在腺苷脱氨酶缺乏症新生儿中的植入。
Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency.
作者信息
Kohn D B, Weinberg K I, Nolta J A, Heiss L N, Lenarsky C, Crooks G M, Hanley M E, Annett G, Brooks J S, el-Khoureiy A
机构信息
Division of Research Immunology/Bone Marrow Transplantation, Childrens Hospital, Los Angeles, University of Southern California School of Medicine 90027, USA.
出版信息
Nat Med. 1995 Oct;1(10):1017-23. doi: 10.1038/nm1095-1017.
Abstract
Haematopoietic stem cells in umbilical cord blood are an attractive target for gene therapy of inborn errors of metabolism. Three neonates with severe combined immunodeficiency were treated by retroviral-mediated transduction of the CD34+ cells from their umbilical cord blood with a normal human adenosine deaminase complementary DNA followed by autologous transplantation. The continued presence and expression of the introduced gene in leukocytes from bone marrow and peripheral blood for 18 months demonstrates that umbilical cord blood cells may be genetically modified with retroviral vectors and engrafted in neonates for gene therapy.
摘要
脐带血中的造血干细胞是先天性代谢缺陷基因治疗的一个有吸引力的靶点。三名患有严重联合免疫缺陷的新生儿接受了如下治疗：从他们的脐带血中提取CD34+细胞，用正常人腺苷脱氨酶互补DNA进行逆转录病毒介导的转导，然后进行自体移植。导入的基因在骨髓和外周血白细胞中持续存在并表达达18个月，这表明脐带血细胞可用逆转录病毒载体进行基因改造，并植入新生儿体内用于基因治疗。
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本文引用的文献
1
Development of gene therapy for immunodeficiency: adenosine deaminase deficiency.免疫缺陷基因治疗的发展：腺苷脱氨酶缺乏症
Pediatr Res. 1993 Jan;33(1 Suppl):S49-53; discussion S53-5. doi: 10.1203/00006450-199305001-00278.
2
Enzyme replacement therapy with polyethylene glycol-adenosine deaminase in adenosine deaminase deficiency: overview and case reports of three patients, including two now receiving gene therapy.聚乙二醇化腺苷脱氨酶用于腺苷脱氨酶缺乏症的酶替代疗法：概述及三例患者病例报告，其中两例患者目前正在接受基因治疗。
Pediatr Res. 1993 Jan;33(1 Suppl):S42-7; discussion S47-8. doi: 10.1203/00006450-199305001-00236.
3
Hematopoietic stem cells engraft in untreated transplant recipients.
Exp Hematol. 1993 Feb;21(2):251-6.
4
Long-term engraftment of normal and post-5-fluorouracil murine marrow into normal nonmyeloablated mice.正常及5-氟尿嘧啶处理后的小鼠骨髓在正常非清髓小鼠体内的长期植入。
Blood. 1993 May 15;81(10):2566-71.
5
Competitive repopulation in unirradiated normal recipients.未受照射的正常受体中的竞争性再增殖
Blood. 1993 May 15;81(10):2473-4.
6
7
Expression of the neomycin-resistance (neo) gene induces alterations in gene expression and metabolism.新霉素抗性（neo）基因的表达会诱导基因表达和代谢的改变。
Hum Gene Ther. 1994 Apr;5(4):449-56. doi: 10.1089/hum.1994.5.4-449.
8
Gene marking to determine whether autologous marrow infusion restores long-term haemopoiesis in cancer patients.基因标记法用于确定自体骨髓输注能否恢复癌症患者的长期造血功能。
Lancet. 1993 Nov 6;342(8880):1134-7. doi: 10.1016/0140-6736(93)92122-a.
9
10
Umbilical cord blood cell transduction by retroviral vectors: preclinical studies to optimize gene transfer.逆转录病毒载体介导的脐血细胞转导：优化基因转移的临床前研究
Blood Cells. 1994;20(2-3):539-43; discussion 544-6.
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