Primary Immunodeficiency Diseases: Current and Emerging Therapeutics.
Primary Immunodeficiency Diseases: Current and Emerging Therapeutics.
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DOI：
10.3389/fimmu.2017.00937
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发表时间：
2017
影响因子：
7.3
通讯作者：
Holland SM
中科院分区：
文献类型：
--
作者：
Marciano BE;Holland SM
原发免疫缺陷疾病是由多种方式影响免疫系统和其他系统的基因缺陷引起的。直到过去几年，除了骨髓移植，治疗在很大程度上是支持性的。然而，免疫生物学、遗传学的最新进展，以及生物修饰剂的发现和商业化的爆炸性增长，极大地改变了临床免疫学的格局和机遇。多发性硬化症患者的治疗选择和预期寿命也大幅提高，这在很大程度上是由于更好地预防和治疗感染以及更好地了解和治疗自身免疫性并发症。随着早期感染相关死亡率的下降，我们应该预料到其他以前没有被认识到的疾病的出现，包括恶性肿瘤和退行性疾病，这些疾病被越来越长的寿命所掩盖。基因组革命已经确定了数百种新的免疫功能障碍的遗传病因，其中许多正在或即将有资格进行靶向治疗。这些新出现的免疫调节剂代表了治疗ID的新选择。
Primary immunodeficiency diseases (PID) result from defects in genes affecting the immune and other systems in many and varied ways. Until the last few years, treatments have been largely supportive, with the exception of bone marrow transplantation. However, recent advances in immunobiology, genetics, and the explosion of discovery and commercialization of biologic modifiers have drastically altered the landscape and opportunities in clinical immunology. Therapeutic options and life expectancy of PID patients have also improved dramatically, in large part as a result of better prevention and treatment of infections as well as better understanding and treatment of autoimmune complications. As early-life infection-related mortality declines we should anticipate the emergence of other conditions that were previously not appreciated, including malignancies and degenerative disorders unmasked by increasing longevity. The genomic revolution has identified literally hundreds of new genetic etiologies of immune dysfunction, many of which are or will soon be eligible for targeted therapies. These emerging immunomodulatory agents represent new therapeutic options in PIDs.
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影响因子：
3.6
作者：
Freeman, Alexandra F.;Marciano, Beatriz E.;Holland, Steven M.
通讯作者：
Holland, Steven M.
DOI：
10.1016/j.jaci.2017.03.049
发表时间：
2018-03
期刊：
The Journal of allergy and clinical immunology
影响因子：
--
作者：
Leiding JW;Okada S;Hagin D;Abinun M;Shcherbina A;Balashov DN;Kim VHD;Ovadia A;Guthery SL;Pulsipher M;Lilic D;Devlin LA;Christie S;Depner M;Fuchs S;van Royen-Kerkhof A;Lindemans C;Petrovic A;Sullivan KE;Bunin N;Kilic SS;Arpaci F;Calle-Martin O;Martinez-Martinez L;Aldave JC;Kobayashi M;Ohkawa T;Imai K;Iguchi A;Roifman CM;Gennery AR;Slatter M;Ochs HD;Morio T;Torgerson TR;Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation and the Primary Immune Deficiency Treatment Consortium
通讯作者：
Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation and the Primary Immune Deficiency Treatment Consortium
影响因子：
8
作者：
Kuijpers, Taco;Lutter, Rene
通讯作者：
Lutter, Rene
影响因子：
27.4
作者：
HENCH, PS;KENDALL, EC;POLLEY, HF
通讯作者：
POLLEY, HF
影响因子：
6.5
作者：
Hennig, Christian;Baumann, Ulrich;Hansen, Gesine
通讯作者：
Hansen, Gesine