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首页> 外文期刊>Expert opinion on biological therapy >Carlecortemcel-l: an ex vivo expanded umbilical cord blood cell graft for allogeneic transplantation.
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Carlecortemcel-l: an ex vivo expanded umbilical cord blood cell graft for allogeneic transplantation.

机译：Carlecortemcel-1：用于异体移植的离体扩增脐带血细胞移植物。

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BACKGROUND: Success of umbilical cord blood transplantation (UCBT) is mostly affected by the cell dose infused and its application is limited by the size of the recipient. For most adults and older children it is not possible to find a single UCB unit large enough for reliable engraftment. One strategy to increase the number of progenitor cells available is ex vivo expansion of the unit. The main challenge of ex vivo expansion systems is how not to deplete the self-renewing cell population by driving them into differentiation into committed progenitors. OBJECTIVE: Copper modulates basic cell functions, such as survival, proliferation, and differentiation. Reduction of cellular copper in ex vivo culture conditions enabled preferential proliferation of early progenitors and increased engraftment capabilities. The result of a Phase I study of carlecortemcel-l, a product derived from ex vivo expansion of UCB progenitors in the presence of a copper chelator and early-acting cytokines, and the study design for the current pivotal study are presented. METHODS: A literature review using PubMed and the investigator's brochure from the manufacturer. CONCLUSIONS: Early results suggest that carlecortemcel-l infusion is safe and may be associated with favorable non-relapse mortality rates. A pivotal global study is currently being conducted to evaluate safety and efficacy of this product from centralized manufacturing facilities.
机译：背景：脐带血移植（UCBT）的成功主要受注入的细胞剂量影响，其应用受到受体大小的限制。对于大多数成人和较大的孩子，不可能找到足够大的单个UCB单元以可靠植入。增加可用祖细胞数量的一种策略是离体扩增该单位。离体扩增系统的主要挑战是如何通过驱使它们分化成定型祖细胞而不耗尽其自身更新细胞。目的：铜调节细胞的基本功能，例如存活，增殖和分化。在离体培养条件下细胞铜的减少使早期祖细胞优先增殖并提高了植入能力。提出了carlecortemcel-1的I期研究结果，carlecortemcel-1是在铜螯合剂和早期作用的细胞因子存在下从UCB祖细胞的体外扩增得到的产物，并提出了当前关键研究的研究设计。方法：使用PubMed和制造商的调查员手册进行文献回顾。结论：早期结果表明，carlecortemcel-1输注是安全的，并且可能与良好的非复发死亡率相关。当前正在进行一项重要的全球研究，以评估集中制造设施中该产品的安全性和有效性。

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