Assessing and advancing the safety of CRISPR-Cas tools: from DNA to RNA editing

J Tao, DE Bauer, R Chiarle - Nature Communications, 2023 - nature.com
CRISPR-Cas gene editing has revolutionized experimental molecular biology over the past
decade and holds great promise for the treatment of human genetic diseases. Here we …

PI3K inhibitors are finally coming of age

B Vanhaesebroeck, MWD Perry, JR Brown… - Nature reviews Drug …, 2021 - nature.com
Abstract Overactive phosphoinositide 3-kinase (PI3K) in cancer and immune dysregulation
has spurred extensive efforts to develop therapeutic PI3K inhibitors. Although progress has …

Targeting PI3K in cancer: mechanisms and advances in clinical trials

J Yang, J Nie, X Ma, Y Wei, Y Peng, X Wei - Molecular cancer, 2019 - Springer
Abstract Phosphatidylinositol-3-kinase (PI3K)/AKT/mammalian target of rapamycin (mTOR)
signaling is one of the most important intracellular pathways, which can be considered as a …

The PI3K pathway in human disease

DA Fruman, H Chiu, BD Hopkins, S Bagrodia… - Cell, 2017 - cell.com
Phosphoinositide 3-kinase (PI3K) activity is stimulated by diverse oncogenes and growth
factor receptors, and elevated PI3K signaling is considered a hallmark of cancer. Many PI3K …

[HTML][HTML] ERα-associated translocations underlie oncogene amplifications in breast cancer

JJK Lee, YL Jung, TC Cheong, J Espejo Valle-Inclan… - Nature, 2023 - nature.com
Focal copy-number amplification is an oncogenic event. Although recent studies have
revealed the complex structure,–and the evolutionary trajectories of oncogene amplicons …

PI3K pathway defects leading to immunodeficiency and immune dysregulation

CJ Nunes-Santos, G Uzel, SD Rosenzweig - Journal of Allergy and Clinical …, 2019 - Elsevier
The phosphatidylinositol 3–kinase (PI3K) signaling pathway is involved in a broad range of
cellular processes, including growth, metabolism, differentiation, proliferation, motility, and …

Frequency and mechanisms of LINE-1 retrotransposon insertions at CRISPR/Cas9 sites

J Tao, Q Wang, C Mendez-Dorantes, KH Burns… - Nature …, 2022 - nature.com
CRISPR/Cas9-based genome editing has revolutionized experimental molecular biology
and entered the clinical world for targeted gene therapy. Identifying DNA modifications …

Treatment of chronic lymphocytic leukemia

JA Burger - New England Journal of Medicine, 2020 - Mass Medical Soc
Chronic Lymphocytic Leukemia Treatment of CLL, the most common leukemia in the West,
has undergone a revolution in the past few years with the development of agents that target …

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

VK Rao, S Webster, VASH Dalm… - Blood, The Journal …, 2017 - ashpublications.org
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 …

Respiratory manifestations of the activated phosphoinositide 3-kinase delta syndrome

AM Condliffe, A Chandra - Frontiers in immunology, 2018 - frontiersin.org
The activated phosphoinositide 3-kinase δ syndrome (APDS), also known as p110δ-
activating mutation causing senescent T cells, lymphadenopathy, and immunodeficiency …