Subject collection: Model Systems in Drug Discovery
- A nonalcoholic fatty liver disease model in human induced pluripotent stem cell-derived hepatocytes, created by endoplasmic reticulum stress-induced steatosis
Summary: Our study demonstrates expanded use of human induced pluripotent stem cell-derived hepatocytes for molecular studies and drug screening, to evaluate new therapeutics with an antisteatotic mechanism of action for nonalcoholic fatty liver disease.
- A Langendorff-like system to quantify cardiac pump function in adult zebrafish
Summary: Here, we describe a new experimental tool to assess contractility of the isolated zebrafish heart perfused in a Langendorff-like system, which will facilitate using zebrafish as a model for human cardiomyopathies.
- Idebenone and coenzyme Q10 are novel PPARα/γ ligands, with potential for treatment of fatty liver diseases
Summary: A zebrafish screen identifies a novel PPARα/γ ligand, idebenone, with potential for treatment of fatty liver diseases, as seen by testing it in a mouse model of type 2 diabetes.
- Modelling glioblastoma tumour-host cell interactions using adult brain organotypic slice co-culture
Editor's choice: A new ex vivo experimental approach to explore how glioblastoma stem cells interact with distinct brain microenvironments, with potential use a as preclinical model using new anticancer agents.
- Curcumin and derivatives function through protein phosphatase 2A and presenilin orthologues in Dictyostelium discoideum
Summary: To unlock the therapeutic potential of curcumin and related compounds, we employ a tractable model system to characterise their cellular and molecular effects and propose novel targets implicated in disease.
- Expanding primary cells from mucoepidermoid and other salivary gland neoplasms for genetic and chemosensitivity testing
Summary: Genetics and therapeutic responses of uncommon neoplasms, including salivary gland cancers, are investigatable through isolation of primary cells from patient material using conditional reprogramming cell technology without compromising pathological diagnosis.
- Early-onset torsion dystonia: a novel high-throughput yeast genetic screen for factors modifying protein levels of torsinAΔE
Summary: A novel high-throughput, automated, genome-wide yeast screen was used to identify genetic modifiers of the steady-state levels of proteins of the dystonia-associated torsinAΔE variant. This article has an associated First Person interview with the first author of the paper as part of the supplementary information.
- Screening in larval zebrafish reveals tissue-specific distribution of fifteen fluorescent compounds
Summary: Fifteen fluorescent compounds have tissue-specific distributions in zebrafish larvae: three anthraquinones were identified as bone dyes in live vertebrates, and two chemotherapeutic drugs were shown to have distinct preferences for the liver.
- Therapeutic strategies for spinal muscular atrophy: SMN and beyond
Summary: Translational research for spinal muscular atrophy (SMA) should address the development of non-CNS and survival motor neuron (SMN)-independent therapeutic approaches to complement and enhance the benefits of CNS-directed and SMN-dependent therapies.
- (CCUG)n RNA toxicity in a Drosophila model of myotonic dystrophy type 2 (DM2) activates apoptosis
Summary: A Drosophila model of myotonic dystrophy type 2 (DM2) recapitulates several features of the human disease, identifies apoptosis as a contributing factor to DM2, and is likely to provide a convenient tool for drug screening.