Subject collection: Model Systems in Drug Discovery
- Urine-sample-derived human induced pluripotent stem cells as a model to study PCSK9-mediated autosomal dominant hypercholesterolemia
Summary: The authors used urine-sample-derived patient-specific human induced pluripotent stem cells to generate hepatocytes carrying gain- or loss-of-function mutations of PCSK9, and mimicking the pathophysiology in vitro.
- T-cell number and subtype influence the disease course of primary chronic lymphocytic leukaemia xenografts in alymphoid mice
Summary: Manipulation of T cells can enhance current CLL xenograft models, expanding their utility for investigation of tumour biology and pre-clinical drug assessment.
- GEMMs as preclinical models for testing pancreatic cancer therapies
Drug Discovery Collection: This Review discusses GEMMs of human pancreatic cancer, particularly focusing on the KPC model, its use in preclinical research, advantages and disadvantages, and its potential for predicting clinical outcomes.
- A rapid in vivo screen for pancreatic ductal adenocarcinoma therapeutics
Editor's choice - Drug Discovery Collection: The study describes a rapid in vivo screen for novel strategies to treat pancreatic ductal adenocarcinoma (PDA).
- Precision-cut kidney slices (PCKS) to study development of renal fibrosis and efficacy of drug targeting ex vivo
Drug Discovery Collection: TGFβ induces renal fibrosis in ex vivo cultured precision-cut kidney slices, which can be attenuated by IFNγ.
- A calixpyrrole derivative acts as an antagonist to GPER, a G-protein coupled receptor: mechanisms and models
Drug Discovery Collection: This paper highlights mechanisms through which a calixpyrrole derivative acts as a newly identified selective antagonist of GPER in different model systems.
- Glycolytic inhibitor 2-deoxyglucose simultaneously targets cancer and endothelial cells to suppress neuroblastoma growth in mice
Drug Discovery Collection: Neuroblastomas are sensitive to treatment with the glycolytic inhibitor 2-deoxyglucose, which can simultaneously target cancer and endothelial cells to suppress tumor growth.
- From drug response profiling to target addiction scoring in cancer cell models
Drug Discovery Collection: The authors take a pan-cancer approach that provides functional insights into molecular vulnerabilities across various cancer subtypes and patient-specific target addiction patterns that could lead to actionable treatment strategies.
- A natural human IgM that binds to gangliosides is therapeutic in murine models of amyotrophic lateral sclerosis
Summary: A single peripheral dose of a recombinant natural human IgM increases lifespan and delays neurological deficits in mouse models of human ALS.