RAG deficiency with ALPS features successfully treated with TCRαβ/CD19 cell depleted haploidentical stem cell transplant.
作者信息
Westermann-Clark Emma, Grossi Alice, Fioredda Francesca, Giardino Stefano, Cappelli Enrico, Terranova Paola, Palmisani Elena, Farmer Jocelyn R, Foldvari Zsofia, Yamazaki Yasuhiro, Faraci Maura, Lanino Edoardo, Notarangelo Luigi D, Dufour Carlo, Ceccherini Isabella, Walter Jolan E, Miano Maurizio
机构信息
University of South Florida Morsani College of Medicine, Department of Internal Medicine, Division of Allergy/Immunology, Tampa, FL, USA.
Medical Genetics Unit, Istituto Giannina Gaslini, Genoa, Italy.
出版信息
Clin Immunol. 2018 Feb;187:102-103. doi: 10.1016/j.clim.2017.10.012. Epub 2017 Nov 20.
Abstract
摘要
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RAG deficiency with ALPS features successfully treated with TCRαβ/CD19 cell depleted haploidentical stem cell transplant.具有自身免疫性淋巴细胞增生综合征（ALPS）特征的重组激活基因（RAG）缺陷通过TCRαβ/CD19细胞去除的单倍体相合干细胞移植成功治疗。
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J Allergy Clin Immunol. 2018 Apr;141(4):1417-1426.e1. doi: 10.1016/j.jaci.2017.07.008. Epub 2017 Aug 3.
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Broad-spectrum antibodies against self-antigens and cytokines in RAG deficiency.RAG缺陷中针对自身抗原和细胞因子的广谱抗体。
J Clin Invest. 2016 Nov 1;126(11):4389. doi: 10.1172/JCI91162.
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Unrelated Hematopoietic Cell Transplantation in a Patient with Combined Immunodeficiency with Granulomatous Disease and Autoimmunity Secondary to RAG Deficiency.一名患有联合免疫缺陷、肉芽肿病及因RAG缺陷继发自身免疫性疾病患者的非亲缘造血干细胞移植
J Clin Immunol. 2016 Oct;36(7):725-32. doi: 10.1007/s10875-016-0326-x. Epub 2016 Aug 18.
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Single-Center Experience of Unrelated and Haploidentical Stem Cell Transplantation with TCRαβ and CD19 Depletion in Children with Primary Immunodeficiency Syndromes.单中心关于原发性免疫缺陷综合征患儿接受TCRαβ和CD19清除的非亲缘及单倍体相合干细胞移植的经验
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Blood. 2014 Jul 31;124(5):822-6. doi: 10.1182/blood-2014-03-563817. Epub 2014 May 28.
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