The case for intrauterine stem cell transplantation
The case for intrauterine stem cell transplantation
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DOI：
10.1016/j.bpobgyn.2012.06.005
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发表时间：
2012-10-01
影响因子：
5.5
通讯作者：
Chan, Jerry K. Y.
中科院分区：
文献类型：
--
作者：
Mattar, Citra N.;Biswas, Arijit;Chan, Jerry K. Y.
单基因疾病集体带来的临床负担需要新的疗法，能够在疾病过程的早期、在发生永久性器官损伤之前有效地实现表型治愈。这对于致命疾病以及以严重残疾且产后治愈前景渺茫为特征的非围产期致命疾病非常重要。在产后治疗仅限于姑息治疗的情况下，宫内干细胞疗法可能有可能阻止早期未受损胎儿的发病机制。宫内干细胞移植已被尝试用于治疗多种疾病，但仅在供体细胞具有竞争优势的免疫缺陷状态下才取得成功。这一令人失望的临床记录需要对改善供体细胞植入的策略进行临床前研究，包括优化供体细胞来源和操纵微环境以促进归巢。本章旨在概述宫内干细胞治疗当前面临的挑战。 (c) 2012 Elsevier Ltd. 保留所有权利。
The clinical burden imposed by the collective group of monogenic disorders demands novel therapies that are effective at achieving phenotypic cure early in the disease process before the development of permanent organ damage. This is important for lethal diseases and also for non-perinatally lethal conditions that are characterised by severe disability with little prospect of postnatal cure. Where postnatal treatments are limited to palliative options, intrauterine stem-cell therapies may offer the potential to arrest pathogenesis in the early undamaged fetus. Intrauterine stem-cell transplantation has been attempted for a variety of diseases, but has only been successful in immune deficiency states in the presence of a competitive advantage for donor cells. This disappointing clinical record requires preclinical investigations into strategies that improve donor cell engraftment, including optimising the donor cell source and manipulating the microenvironment to facilitate homing. This chapter aims to outline the current challenges of intrauterine stem-cell therapy. (c) 2012 Elsevier Ltd. All rights reserved.