Subject collection: Model Systems in Drug Discovery
- Using systems medicine to identify a therapeutic agent with potential for repurposing in inflammatory bowel disease
Summary: Using state-of-the-art in silico drug discovery and real-time live-cell imaging techniques, we identify clarithromycin as a drug with potential for repositioning for inflammatory bowel disease.
- An HIV-Tat inducible mouse model system of childhood HIV-associated nephropathy
Summary: The development of a new inducible mouse model system of childhood HIV-associated nephropathy demonstrated that HIV-Tat plays a critical role in this disease, acting in synergy with other HIV-1 genes and heparin-binding cytokines.
- Cellular and animal models for facioscapulohumeral muscular dystrophy
Summary: Owing to its complex etiology and the toxicity of DUX4, modeling facioscapulohumeral muscular dystrophy (FSHD) is uniquely challenging. Here, we review the approaches that overcame these difficulties to develop highly relevant FSHD models.
- Modeling neurodegeneration in Caenorhabditis elegans
Summary: While unsurpassed as an experimental system for fundamental biology, Caenorhabditis elegans remains undervalued for its translational potential. Here, we highlight significant outcomes from, and resources available for, C. elegans-based research into neurodegenerative disorders.
- Pre-existing antibody-mediated adverse effects prevent the clinical development of a bacterial anti-inflammatory protein
Summary: Chemotaxis inhibitory protein of Staphylococcus aureus dampens C5a-mediated responses in human-C5aR1 knock-in mice. Feasibility studies in a human phase I trial failed due to high levels of existing natural antibodies causing adverse effects.
- Integrating fish models in tuberculosis vaccine development
Summary: In this Review, we discuss how zebrafish (Danio rerio) and other fish models can complement the more traditional mammalian models in the development of novel vaccines against tuberculosis.
- Neuromuscular disease modeling on a chip
Summary: Modeling neuromuscular diseases is challenging due to their complex etiology and pathophysiology. Here, we review the cell sources and tissue-engineering procedures that are being integrated as emerging neuromuscular disease models.
- Precision-based modeling approaches for necrotizing enterocolitis
Summary: Much of our understanding of necrotizing enterocolitis (NEC) pathogenesis has been achieved through animal models. Here, we discuss the development of advanced precision-based models to improve outcomes for patients with NEC.
- Mouse models for muscular dystrophies: an overview
Summary: This Review and its accompanying comprehensive table summarize the most commonly used mouse models for a subset of highly studied muscular dystrophies.
- Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT)
Summary: Rigorous preclinical efficacy studies de-risk clinical trials in neuromuscular diseases. This Review summarizes the impact of expert panel advice on translational studies.