Milestones in Personalized Medicine in Pemphigus and Pemphigoid.
Milestones in Personalized Medicine in Pemphigus and Pemphigoid.
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DOI：
10.3389/fimmu.2020.591971
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发表时间：
2020
影响因子：
7.3
通讯作者：
Ludwig RJ
中科院分区：
文献类型：
--
作者：
Bieber K;Kridin K;Emtenani S;Boch K;Schmidt E;Ludwig RJ
天疱疮和类天疱疮疾病是自身免疫性大疱性疾病，其特征是由针对皮肤和/或粘膜粘附分子的自身抗体引起的。个性化医疗是一种新的医疗模式，它将患者分为不同的群体，旨在根据个体患者预测的反应或风险因素量身定制医疗决策、实践和干预措施。通过验证这些疾病的自身免疫性发病机制以及鉴定和克隆几种天疱疮/类天疱疮自身抗原，实现了天疱疮和类天疱疮个体化治疗的重要里程碑。后者已成为目前的基础，分子为基础的诊断，允许区分大约十几天疱疮和类天疱疮实体。自身抗原特异性B细胞消耗策略的出现进一步强调了自身抗原鉴定在天疱疮/类天疱疮中的重要性。为了实现这一目标，通过产生嵌合自身抗原受体（CAAR） T细胞，采用了用于治疗某些血液系统恶性肿瘤的嵌合抗原受体（CAR） T细胞技术。除了在天疱疮和类天疱疮个体化医疗方面这些更基础的科学驱动的里程碑之外，仔细的临床观察和流行病学再次为个体化医疗做出贡献。在类天疱疮中明确不同的临床表型的鉴定，如非炎症性和格列汀相关的大疱性类天疱疮，体现了后者的突出实例。我们在这里回顾这些令人兴奋的发展在基础，转化，临床和流行病学研究天疱疮和类天疱疮。总的来说，我们的目标是吸引更多的研究人员和临床医生到这个非常有趣和充满活力的研究领域。
Pemphigus and pemphigoid diseases are autoimmune bullous diseases characterized and caused by autoantibodies targeting adhesion molecules in the skin and/or mucous membranes. Personalized medicine is a new medical model that separates patients into different groups and aims to tailor medical decisions, practices, and interventions based on the individual patient`s predicted response or risk factors. An important milestone in personalized medicine in pemphigus and pemphigoid was achieved by verifying the autoimmune pathogenesis underlying these diseases, as well as by identifying and cloning several pemphigus/pemphigoid autoantigens. The latter has become the basis of the current, molecular-based diagnosis that allows the differentiation of about a dozen pemphigus and pemphigoid entities. The importance of autoantigen-identification in pemphigus/pemphigoid is further highlighted by the emergence of autoantigen-specific B cell depleting strategies. To achieve this goal, the chimeric antigen receptor (CAR) T cell technology, which is used for the treatment of certain hematological malignancies, was adopted, by generating chimeric autoantigen receptor (CAAR) T cells. In addition to these more basic science-driven milestones in personalized medicine in pemphigus and pemphigoid, careful clinical observation and epidemiology are again contributing to personalized medicine. The identification of clearly distinct clinical phenotypes in pemphigoid like the non-inflammatory and gliptin-associated bullous pemphigoid embodies a prominent instance of the latter. We here review these exciting developments in basic, translational, clinical, and epidemiological research in pemphigus and pemphigoid. Overall, we hereby aim to attract more researchers and clinicians to this highly interesting and dynamic field of research.
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影响因子：
64.5
作者：
AMAGAI, M;KLAUSKOVTUN, V;STANLEY, JR
通讯作者：
STANLEY, JR
影响因子：
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作者：
Giusti D;Gatouillat G;Le Jan S;Plée J;Bernard P;Antonicelli F;Pham BN
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Pham BN
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作者：
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通讯作者：
Hertl, Michael
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4.8
作者：
Chernyavsky, Alex;Amber, Kyle T.;Grando, Sergei A.
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Grando, Sergei A.