HUMAN UMBILICAL CORD BLOOD CELL BANKING AND EXPANSION

人脐带血细胞储存和扩增

• 批准号: 6302369
• 负责人: STEVEN EMERSON
• 金额: $ 40.59万
• 依托单位: UNIVERSITY OF PENNSYLVANIA
• 依托单位国家: 美国
• 财政年份: 2000
• 资助国家: 美国
• 起止时间: 2000-02-15 至 2001-12-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6302369
• 关键词: African American; SCID mouse; bioreactors; blood banks; blood donor; blood transfusion; blood treatment; bone marrow transplantation; cell growth regulation; cell transplantation; clinical research; cryopreservation; hematopoietic stem cells; human subject; mass tissue /cell culture; sickle cell anemia; umbilical cord

Recent progress in clinical and laboratory investigations has shown that Human Umbilical Cord Blood (UCB) contains cells capable of sustained hematopoietic engraftment, and therefore UCB has vast potential as a source of hematopoietic stem cells for allogeneic and autologous bone marrow transplantation. UCB is particularly attractive because of the possibility that its use might decrease the incidence and severity of graft versus host disease, and thereby decrease overall post-transplant complications. UCB offers major theoretical benefits as a treatment for hemoglobinopathies, where the high disease incidence in non-Caucasian patients makes matched unrelated donor bone marrow searches problematic at present. Several issues need to be addressed however, before UCB can be routinely used as a transfusion product for stern cell support, including increasing the number of transplantable stem cells available from a given volume of UCB, and developing a UCB bank that maximizes the chances of banked UCB units to be used for clinical transplants. It is the intent of this project to attack these issues, with the goal of transforming UCB from an exotic research field to a routine blood bank transfusion therapy that will be of decisive benefit to common, serious diseases such as sickle cell anemia. In particular, we plan to develop a facility that will be tailored for correction of homozygous Sickle Cell Anemia by human umbilical cord blood cell transplantation. The program will both target our loco-regional population, heavily represented by African-Americans, and will serve as a national resource as well. To achieve this goal, we propose two Specific Aims: 1) To develop a HUCB bank targeted at the African-American population, emphasizing application towards correction of Sickle Cell Disease, and 2) To explore and develop techniques of HUCB stem cell expansion. Achieving these two goals should make major studies towards enabling the widespread cure of SS disease through HUCB transplantation.

临床和实验室研究的最新进展表明 人脐带血 (UCB) 含有能够持续 造血移植，因此 UCB 作为来源具有巨大的潜力 用于同种异体和自体骨髓的造血干细胞 移植。 UCB 特别有吸引力，因为它有可能 它的使用可能会降低移植物抗宿主病的发生率和严重程度 疾病，从而减少整体移植后并发症。 联合银行 作为血红蛋白病的治疗提供了重大的理论益处， 非白人患者的高发病率与此相匹配 目前，无关的供体骨髓搜索存在问题。 然而，在常规 UCB 之前，需要解决几个问题 用作干细胞支持的输血产品，包括增加 给定体积的可移植干细胞的数量 UCB，并开发一家 UCB 银行，最大限度地提高 UCB 存入银行的机会 用于临床移植的单位。 正是这个目的 项目旨在解决这些问题，目标是将 UCB 从 常规血库输血疗法的奇异研究领域 对镰状细胞病等常见严重疾病具有决定性益处 贫血。 特别是，我们计划开发一个量身定制的设施 用于通过人脐带矫正纯合镰状细胞贫血症 血细胞移植。 该计划将针对我们的本地区域 人口中以非裔美国人为主，并将作为 也是国家资源。 为实现这一目标，我们提出两个具体方案 目标： 1) 开发针对非裔美国人的 HUCB 银行， 强调纠正镰状细胞病的应用，以及 2) 探索和开发HUCB干细胞扩增技术。 实现 这两个目标应该进行重大研究，以实现广泛的 通过HUCB移植治愈SS病。