Effective “activated PI3Kδ syndrome”–targeted therapy with the PI3Kδ inhibitor leniolisib

Pathogenic gain-of-function variants in the genes encoding phosphoinositide 3-kinase δ (PI3Kδ) lead to accumulation of transitional B cells and senescent T cells, lymphadenopathy, and immune deficiency (activated PI3Kδ syndrome [APDS]). Knowing the genetic etiology of APDS afforded us the opportunit...

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書誌詳細
出版年:Blood
主要な著者: Rao, V. Koneti, Webster, Sharon, Dalm, Virgil A. S. H., Šedivá, Anna, van Hagen, P. Martin, Holland, Steven, Rosenzweig, Sergio D., Christ, Andreas D., Sloth, Birgitte, Cabanski, Maciej, Joshi, Aniket D., de Buck, Stefan, Doucet, Julie, Guerini, Danilo, Kalis, Christoph, Pylvaenaeinen, Ilona, Soldermann, Nicolas, Kashyap, Anuj, Uzel, Gulbu, Lenardo, Michael J., Patel, Dhavalkumar D., Lucas, Carrie L., Burkhart, Christoph
フォーマット: Artigo
言語:Inglês
出版事項: American Society of Hematology 2017
主題:
Immunobiology and Immunotherapy
オンライン･アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC5701526/
https://ncbi.nlm.nih.gov/pubmed/28972011
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2017-08-801191
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