Stem Cell Core

干细胞核心

• 批准号: 7784220
• 负责人: Brian P Sorrentino
• 金额: $ 35.27万
• 依托单位: ST. JUDE CHILDREN'S RESEARCH HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-09-01 至 2015-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7784220
• 关键词: Antibodies; Biological Assay; Blood Cells; Blood specimen; Bone Marrow; Bone Marrow Cells; Bone Marrow Transplantation; Budgets; CD34 gene; CSF3 gene; Cell Therapy; Cell physiology; Cells; Clinical; Clinical Trials; Development; Enrollment; Flow Cytometry; Funding; Gene Transfer; Hematopoietic; Hematopoietic stem cells; Human; Injection of therapeutic agent; Laboratories; Macaca nemestrina; Modeling; Mus; Patients; Persons; Physician Executives; Procedures; Process; Protocols documentation; Research Ethics Committees; Resources; Saint Jude Children' s Research Hospital; Serum-Free Culture Media; Services; Sickle Cell; Stem cells; System; Testing; Time; United States National Institutes of Health; Wing; Work; base; cellular transduction; clinical material; cytokine; genetically modified cells; in vitro Assay; nonhuman primate; peripheral blood; pre-clinical; preclinical study; programs; reconstitution; research study; transduction efficiency; vector; volunteer

Stem Cell Core B is directed by Drs. Sorrentino and Persons and provides for stem cell procurement, processing, and manipulation for both the preclinical and clinical trial procedures within this application. Dr. Persons will direct the preclinical activities in this Core which include purification and transduction of nonhuman primate cells as outlined in Project 1 as well as purification of mouse hematopoietic stem cells from murine SCD and WAS models in support of Projects 1 and 2. The Core will continue to define optimal lentiviral transduction conditions for CD34+ cells obtained from human bone marrow and G-CSF mobilized peripheral blood cells from normal volunteers. Transduction efficiencies will be determined using standard in vitro assays such as flow cytometry and CFU-C assays and also by reconstitution experiments in immunodeficient NOG mice. In addition to these preclinical and developmental functions, Core B will provide for the processing and transduction of clinical material obtained from St. Jude patients and used for the WAS clinical trial proposed in Project 2 and the LVXSCID trials in Project 3. These activities will be directed by Dr. Sorrentino. Patients treated on the LVSCID-OC trial at NIH will continue to use existing resources at the NIH clinical center under the direction of Dr. Malech and his colleagues and no funds are requested for these activities. For St. Jude patients, mobilized peripheral blood cells or bone marrow cells from patients will be collected, CD34+ cells will be obtained using a certified procedure based on the CliniMacs apparatus, cells will be transduced with certified vector supernatants prepared in our GMP facility, and processed cells will be prepared for injection into the patient. All of these clinical manipulations will be done in the St. Jude Human Applications Laboratory (HAL), which is an established clinical laboratory that supports all activities that are ongoing in the Division of Bone Marrow Transplant and Cell Therapy. Because of this expanded scope and emphasis on clinical trials, the activities and budget for the Stem Cell Core have increased from the past funding period.

干细胞核B由DRS指导。 Sorrentino和人，并提供干细胞采购， 该应用程序中的临床前和临床试验程序的处理以及操纵。博士 人员将指导该核心的临床前活动，包括纯化和转导非人类 在项目1中概述的灵长类动物细胞以及小鼠造血干细胞的纯化 Murine SCD，是支持项目1和2的模型。核心将继续定义最佳 从人骨髓和G-CSF中获得的CD34+细胞的慢病毒转导条件动员 正常志愿者的外周血细胞。转导效率将使用标准确定 体外测定，例如流式细胞术和CFU-C测定法，以及通过重新建立实验 免疫缺陷的NOG小鼠。除了这些临床前和发展功能外，核心B还将提供 用于从圣裘德患者获得的临床材料的处理和转导，用于 在项目2和项目3中的LVXSCID试验中提出的临床试验。这些活动将由博士指导。 索伦蒂诺。 NIH在LVSCID-OC试验中接受治疗的患者将继续使用NIH现有资源 在Malech博士及其同事的指导下，临床中心没有要求这些资金 活动。对于圣裘德患者，动员的外周血细胞或患者的骨髓细胞将是 收集的，将使用基于Clinimacs设备的认证程序获得CD34+细胞 将用在我们的GMP设施中准备的经过认证的矢量上清液转导，并且处理后的细胞将是 准备向患者注射。所有这些临床操作都将在圣裘德人类中进行 应用实验室（HAL），这是一个既定的临床实验室，支持所有活动 骨髓移植和细胞疗法的分裂中。由于这个扩大范围， 强调临床试验，干细胞核心的活动和预算从过去增加 资金期。