Proliferative Switch for Genetically Modified Cells

转基因细胞的增殖转换

• 批准号: 6399264
• 负责人: C. Anthony Blau
• 金额: $ 27.26万
• 依托单位: UNIVERSITY OF WASHINGTON
• 依托单位国家: 美国
• 财政年份: 1997
• 资助国家: 美国
• 起止时间: 1997-08-10 至 2005-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6399264
• 关键词: CD34 molecule; Retroviridae; animal genetic material tag; bone marrow transplantation; cell proliferation; developmental genetics; developmental immunology; dimer; dogs; flow cytometry; gene induction /repression; gene targeting; genetically modified animals; growth factor receptors; hematopoiesis; hematopoietic stem cells; immunogenetics; immunopharmacology; laboratory mouse; megakaryocytes; polymerase chain reaction; thrombocytopenia; thrombopoietic factor; tissue /cell culture; transfection /expression vector

DESCRIPTION (provided by applicant): This application is a request for renewal of our R01 entitled: "A proliferative switch for genetically modified cells," which was funded beginning in August 1997. The stated goal of that application was "to apply newly developed technology to the expansion of genetically modified hemopoietic cells in vitro or in vivo." We have achieved that goal. Our approach uses a derivative of the thrombopoietin receptor (mpl) that delivers a conditional growth signal in response to a drug called a chemical inducer of dimerization (CID). In specific aim 1 we will humanize the mpl vector. In specific aim 2 we will test our humanized vector in normal mice and in a mouse model of mpl deficiency. In specific aim 3 we will test the humanized vector in a canine model. in specific aim 4 we will test the humanized vector in hemopoietic cells taken from patients with mpl deficiency.

描述（由申请人提供）：此申请是续订的请求 我们的R01的标题为：“转基因细胞的增殖开关”，” 这是从1997年8月开始的。该申请的既定目标 是“将新开发的技术应用于基因扩展 在体外或体内修饰的血压细胞。“我们实现了这个目标。 我们的方法使用血小板素受体（MPL）的衍生物 发出有条件的生长信号，以响应一种称为化学的药物 二聚化诱导者（CID）。在特定目标1中，我们将人性化MPL 向量。在特定目标2中，我们将在正常小鼠中测试我们的人源化向量，并 在MPL缺乏症的鼠标模型中。在特定目标3中，我们将测试 犬模型中的人源化载体。在特定目标4中，我们将测试 从具有MPL缺乏的患者中吸收的血压细胞中的人源化载体。