胎儿移植研究在亨廷顿氏病中是否仍然是合理的?

Q4 Neuroscience
Neuronal signaling Pub Date : 2021-12-13 eCollection Date: 2021-12-01 DOI:10.1042/NS20210019
Oliver J M Bartley, Mariah J Lelos, William P Gray, Anne E Rosser
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引用次数: 2

摘要

早期中枢神经系统移植研究使用胎儿源性细胞产品,为临床前研究和早期临床试验的功能恢复提供了基础证据。然而,人们很快认识到,胎儿组织的实际限制使其不适合广泛的临床应用。从那以后,大量的研究工作转向了从多能干细胞(PSCs)中产生靶细胞表型,现在有一些出版物详细介绍了与亨廷顿病(HD)移植相关的多能干细胞衍生产品的分化和特征。鉴于这一进展,我们想知道胎儿组织移植在HD研究中是否仍然是合理的。我们认为(i)目前从PSCs中产生准确分化的靶细胞的程度仍不清楚,目前使它们不适合研究更广泛的中枢神经系统移植问题;(ii)胎儿源性细胞在临床前研究中仍然是一个有价值的工具,可以促进我们对哪些产品产生功能性纹状体移植物的理解,并作为进一步改进psc源性产品的参考;(iii)在psc衍生产品准备用于人体试验之前,重要的是继续使用胎儿细胞来收集临床证据,证明移植是HD的可行选择,并利用这个机会优化实际参数(如试验设计、临床实践和递送策略),为未来的psc衍生产品铺平道路。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Do foetal transplant studies continue to be justified in Huntington's disease?

Early CNS transplantation studies used foetal derived cell products to provide a foundation of evidence for functional recovery in preclinical studies and early clinical trials. However, it was soon recognised that the practical limitations of foetal tissue make it unsuitable for widespread clinical use. Considerable effort has since been directed towards producing target cell phenotypes from pluripotent stem cells (PSCs) instead, and there now exist several publications detailing the differentiation and characterisation of PSC-derived products relevant for transplantation in Huntington's disease (HD). In light of this progress, we ask if foetal tissue transplantation continues to be justified in HD research. We argue that (i) the extent to which accurately differentiated target cells can presently be produced from PSCs is still unclear, currently making them undesirable for studying wider CNS transplantation issues; (ii) foetal derived cells remain a valuable tool in preclinical research for advancing our understanding of which products produce functional striatal grafts and as a reference to further improve PSC-derived products; and (iii) until PSC-derived products are ready for human trials, it is important to continue using foetal cells to gather clinical evidence that transplantation is a viable option in HD and to use this opportunity to optimise practical parameters (such as trial design, clinical practices, and delivery strategies) to pave the way for future PSC-derived products.

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CiteScore
4.60
自引率
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