Sickle Cell Disease Stem Cell Repository

镰状细胞病干细胞储存库

• 批准号: 10018999
• 负责人: JOHN P MANIS
• 金额: $ 120.89万
• 依托单位: BOSTON CHILDREN'S HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-24 至 2024-09-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10018999
• 关键词: Blood Component Removal; CD34 gene; Cells; Collection; Freezing; Institutional Review Boards; Magnetism; Patients; Peripheral Blood Stem Cell; Pharmaceutical Preparations; Phase; Protocols documentation; Research Personnel; Resources; Ships; Sickle Cell Anemia; Stem cells; Training and Education; Validation; peripheral blood; recruit; repository; therapy development; volunteer

We plan to recruit volunteers with SCD as healthy donors for collection of peripheral blood stem cells by apheresis. Volunteers will be treated with plerixafor, a drug that we and others have shown to mobilize peripheral blood (PB) CD34+ cells safely in SCD patients. The collected stem cells will be purified by magnetic separation using CD34 beads, validated, frozen, and then provided as a resource to investigators working on developing therapies for SCD.

我们计划招募患有SCD的志愿者作为健康捐献者进行外周血干细胞采集 细胞分离术。志愿者将接受普乐沙福治疗，这是我们和其他人拥有的一种药物 研究表明，它可以在 SCD 患者中安全地动员外周血 (PB) CD34+ 细胞。收集到的 干细胞将使用 CD34 磁珠通过磁力分离进行纯化、验证、冷冻，然后 作为资源提供给致力于开发 SCD 疗法的研究人员。