Hematopoietic stem cell transplantation for activated phosphoinositide 3-kinase δ syndrome: Who, when, and how?
作者信息
Notarangelo Luigi D
机构信息
Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md.
出版信息
J Allergy Clin Immunol. 2019 Jan;143(1):91-93. doi: 10.1016/j.jaci.2018.08.039. Epub 2018 Sep 12.
Abstract
摘要
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本文引用的文献
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Hematopoietic stem cell transplantation for progressive combined immunodeficiency and lymphoproliferation in patients with activated phosphatidylinositol-3-OH kinase δ syndrome type 1.用造血干细胞移植治疗 1 型活化磷脂酰肌醇-3-羟激酶 δ 综合征患者的进行性联合免疫缺陷和淋巴组织增生。
J Allergy Clin Immunol. 2019 Jan;143(1):266-275. doi: 10.1016/j.jaci.2018.04.032. Epub 2018 May 18.
2
Disease Evolution and Response to Rapamycin in Activated Phosphoinositide 3-Kinase δ Syndrome: The European Society for Immunodeficiencies-Activated Phosphoinositide 3-Kinase δ Syndrome Registry.激活的磷酸肌醇 3-激酶 δ 综合征中疾病的演变和雷帕霉素的反应：欧洲免疫缺陷学会-激活的磷酸肌醇 3-激酶 δ 综合征登记处。
Front Immunol. 2018 Mar 16;9:543. doi: 10.3389/fimmu.2018.00543. eCollection 2018.
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Activated PI3 Kinase Delta Syndrome: From Genetics to Therapy.活化的 PI3Kδ 综合征：从遗传学治疗。
Front Immunol. 2018 Feb 27;9:369. doi: 10.3389/fimmu.2018.00369. eCollection 2018.
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Effective "activated PI3Kδ syndrome"-targeted therapy with the PI3Kδ inhibitor leniolisib.使用PI3Kδ抑制剂来那度胺进行有效的“活化PI3Kδ综合征”靶向治疗。
Blood. 2017 Nov 23;130(21):2307-2316. doi: 10.1182/blood-2017-08-801191. Epub 2017 Sep 29.
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Phosphatidylinositol 3-kinase δ blockade increases genomic instability in B cells.磷脂酰肌醇3激酶δ阻断增加B细胞中的基因组不稳定性。
Nature. 2017 Feb 23;542(7642):489-493. doi: 10.1038/nature21406. Epub 2017 Feb 15.
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Hematopoietic stem cell transplant in patients with activated PI3K delta syndrome.伴有活化PI3Kδ综合征患者的造血干细胞移植
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7
PI3Kδ and primary immunodeficiencies.磷脂酰肌醇-3激酶δ与原发性免疫缺陷
Nat Rev Immunol. 2016 Nov;16(11):702-714. doi: 10.1038/nri.2016.93. Epub 2016 Sep 12.
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Clinical spectrum and features of activated phosphoinositide 3-kinase δ syndrome: A large patient cohort study.活化磷脂酰肌醇3-激酶δ综合征的临床谱和特征：一项大型患者队列研究。
J Allergy Clin Immunol. 2017 Feb;139(2):597-606.e4. doi: 10.1016/j.jaci.2016.06.021. Epub 2016 Jul 16.
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Clinical and immunologic phenotype associated with activated phosphoinositide 3-kinase δ syndrome 2: A cohort study.激活的磷酸肌醇 3-激酶 δ 综合征 2 相关的临床和免疫表型：一项队列研究。
J Allergy Clin Immunol. 2016 Jul;138(1):210-218.e9. doi: 10.1016/j.jaci.2016.03.022. Epub 2016 Apr 21.
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Dominant-activating germline mutations in the gene encoding the PI(3)K catalytic subunit p110δ result in T cell senescence and human immunodeficiency.编码 PI(3)K 催化亚基 p110δ 的基因中的显性激活种系突变导致 T 细胞衰老和人类免疫缺陷。
Nat Immunol. 2014 Jan;15(1):88-97. doi: 10.1038/ni.2771. Epub 2013 Oct 28.
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