RETROVIRAL TRANSFER OF CDNA FOR GLUCOCEREBROSIDASE INTO STEM CELLS

葡萄糖脑苷脂酶 cDNA 的逆转录病毒转移至干细胞

• 批准号: 6274840
• 负责人: Donald B Kohn
• 金额: $ 3.67万
• 依托单位: UNIVERSITY OF SOUTHERN CALIFORNIA
• 依托单位国家: 美国
• 财政年份: 1997
• 资助国家: 美国
• 起止时间: 1997-12-01 至 1998-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6274840
• 关键词: CD34 molecule; Gaucher's disease; Retroviridae; bone marrow; cell transplantation; clinical research; colony stimulating factor; complementary DNA; cytotoxicity; enzyme activity; gene expression; gene targeting; glucosylceramidase; hematopoietic stem cells; human subject; human therapy evaluation; macrophage; transfection; transfection /expression vector

Patients with Gaucher disease suffer from a lack of functional glucocerebrosidase enzyme (Gc). This study is designed to determine if cDNA encoding normal Gc can be introduced into macrophage precursors using a retroviral vector. CD34+ cells obtained from GCSF mobilized peripheral blood stem cells or from bone marrow will be transduced ex vivo. These transduced cells will be reinfused into the patient who will be monitored for toxcities as well as evidence of successful transfer and expression.

高彻氏病患者缺乏功能性 葡萄糖脑溴糖苷酶（GC）。这项研究旨在确定是否 可以使用编码正常GC的cDNA使用使用 逆转录病毒载体。从GCSF获取的CD34+细胞动员周围 血干细胞或骨髓将被转导离体。 这些 转导的细胞将被重新融入将受到监测的患者 用于毒品以及成功转移和表达的证据。