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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Udgivet i:Blood
Main Authors: 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
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Hematology 2017
Fag:
Immunobiology and Immunotherapy
Online adgang: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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