Stem cell transplantation for the treatment of immunodeficiency in children: current status and hopes for the future
Stem cell transplantation for the treatment of immunodeficiency in children: current status and hopes for the future
复制标题
干细胞移植治疗儿童免疫缺陷：现状与未来希望
DOI：
10.1586/1744666x.2016.1150177
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发表时间：
2016
影响因子：
4.4
通讯作者：
P. Veys
中科院分区：
文献类型：
--
作者：
C. Booth;Juliana M. F. Silva;P. Veys
摘要原发性免疫缺陷是一种罕见的影响免疫功能的遗传性疾病，如果不进行治疗，可能会危及生命。造血干细胞移植(HSCT)为许多此类疾病提供了一种治疗方法，对于缺乏合适供者的患者，基因治疗越来越多地被用作替代治疗策略。早期诊断、改善的支持性护理以及基因和细胞疗法的进步导致了存活率的提高和生活质量的改善。这篇综述描述了目前用于改善PID预后的策略，重点介绍了HSCT、基因和细胞治疗的新进展。我们还应对与新生儿筛查(NBS)计划和通过改进诊断技术发现的新突变相关的挑战。
ABSTRACT Primary immunodeficiencies (PID) are rare inherited disorders affecting immune function and can be life-threatening if not treated. Haematopoietic stem cell transplantation (HSCT) offers a curative approach for many of these disorders and gene therapy is increasingly used as an alternative therapeutic strategy for patients lacking a suitable donor. Early diagnosis, improved supportive care and advances in gene and cell therapies have resulted in increased survival rates and improved quality of life. This review describes current strategies employed to improve outcomes in PID, focusing on new developments in HSCT, gene and cell therapy. We also address the challenges associated with newborn screening (NBS) programmes and novel mutations identified through improved diagnostic technology.
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影响因子：
20.3
作者：
Leen, Ann M.;Bollard, Catherine M.;Heslop, Helen E.
通讯作者：
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20.3
作者：
Zhou, Xiaoou;Di Stasi, Antonio;Dotti, Gianpietro
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Dotti, Gianpietro
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20.3
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20.3
作者：
Moratto, Daniele;Giliani, Silvia;Notarangelo, Luigi D.
通讯作者：
Notarangelo, Luigi D.