Article
Effective "activated PI3Kδ syndrome"-targeted therapy with the PI3Kδ inhibitor leniolisib.
Blood - 23 Nov 2017
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
Abstract excerpt
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 opportunity to explore PI3Kδ inhibition as a precision-medicine therapy. Here, we report in vitro and in vivo effects of...
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