Stem Cell Core

干细胞核心B由Sorrentino博士和Person指导，并提供干细胞采购， 此应用程序中的临床前和临床试验程序的处理和操作。Dr。 个人将在这个核心指导临床前活动，包括非人类的纯化和转导 项目1中概述的灵长类细胞以及小鼠造血干细胞的纯化 支持项目1和2的小鼠SCD和WAS模型。核心将继续定义最优 慢病毒转导人骨髓CD34+细胞及动员G-CSF的条件 来自正常志愿者的外周血细胞。转导效率将使用标准的 体外检测，如流式细胞术和CFU-C检测，也通过重建实验 免疫缺陷的NOG小鼠。除了这些临床前和开发功能外，Core B还将提供 用于处理和传输从圣犹大患者那里获得的临床材料，并用于 项目2中建议的临床试验和项目3中的LVXSCID试验。 索伦蒂诺。在NIH接受LVSCID-OC试验治疗的患者将继续使用NIH现有的资源 由Malech博士和他的同事指导的临床中心，不需要为这些项目申请资金 活动。对于圣犹大患者，从患者身上动员的外周血细胞或骨髓细胞将 收集到的CD34+细胞将使用基于CliniMacs设备的认证程序获得，细胞 将与我们的GMP设施中准备的经认证的载体上清液进行转导，处理后的细胞将 准备注射到病人体内。所有这些临床操作都将在圣犹大人类医院完成 应用实验室(HAL)，这是一个已建立的临床实验室，支持以下所有活动 骨髓移植和细胞治疗分部正在进行中。由于这种扩展的范围和 重视临床试验，干细胞核心的活动和预算比过去有所增加 资助期。

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.