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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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Detalhes bibliográficos
Publicado no: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
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2017
Assuntos:
Acesso em linha: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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