Getting “under the skin” of frontotemporal dementia
Researchers at the University of East Finland have been using skin cells to investigate pathological hallmarks in frontotemporal dementia patients.
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“Noisy” gene expression may help improve stem cell therapies
Researchers have identified a process that amplifies changes in gene expression, which could be harnessed to accelerate stem cell differentiation.
Exclusive report: AI & Informatics: Drug discovery and development
In this original report, find an in-depth analysis of AI and informatics within imaging, synthetic biology, drug screening and drug design. Featured interviews with experts from AstraZeneca, Auransa, PolarisQB and Chalmers University of Technology.
Spatial genomic and transcriptomic imaging in drug discovery
In this interview, read about the compilation of a new high-resolution cell atlas of the mouse brain using spatial genomics and transcriptomics.
New models for intranasal drug delivery
In this article, Dr Jon Volmer and Dr Jon Lenn discuss a new approach to formulation models to test drug delivery based on reconstituted nasal epithelium.
Lentiviral vectors: the key to cancer treatments that CAR T-cell therapy cannot reach?
Professor Christian Brechot explains why lentiviral vectors could serve as an effective tool for treating a wide range of cancers and could be used for vaccines.
Recognition of phosphohistidine-containing peptides by antibodies
In this article, Dr Rajasree Kalagiri explains research into the use of monoclonal antibodies for the differential recognition of phosphohistidine-containing peptides.
How engineered RNA binding proteins delivered in vivo by gene therapy could treat myotonic dystrophy
In this article, Dr Jim Burns discusses promising pre-clinical results of how a new platform could treat the root cause of many devastating genetic diseases including myotonic dystrophy type 1.
Why the system for biospecimen procurement is broken
Dr Robert Hewitt explains why the need of biospecimen brokers to protect their commercially-sensitive information can result in significant issues for industry end-users and the results of their research.
Transcriptional reprogramming rescues vascular fate of misidentified pluripotent cell‑derived epithelial cells
A team of scientists has found that a type of cell derived from human stem cells and widely used for brain research and drug development may have been leading researchers astray for years. Here, Dr Raphaël Lis from Weill Cornell Medicine explains how forcing the activity of three known endothelial cell transcription…
Drug development in stem cell-derived liver organoid models
To ensure that new drugs are effective and have as little toxicity as possible, they first need to be tested in model systems before entering clinical trials. In this Q&A, Dr Takanori Takebe outlines his research into liver organoids derived from stem cells that can be used to test new…
Making cell culture models more physiologically relevant
A major limitation in drug development is the occurrence of drug-candidate toxicity during clinical research. This may occur because tumour-derived cell lines are limited as a pre-clinical model – in part because of an altered metabolic poise. A recent study has revealed a profound plasticity in gene expression and metabolic poise that can…
Novel vectors for more efficient and less risky ocular gene therapy
Inherited blindness is the leading cause of vision loss in the working populations. Recently, the first gene therapy for the treatment of retinal dystrophy caused by mutations in the RPE65 gene became available. This gene therapy is based on adeno-associated virus (AAV) vectors injected under the retina. Subretinal injection is…
Using cholangiocyte organoids to regenerate human bile ducts
A team of researchers has shown that injection of cholangiocyte organoids in human livers ex vivo can repair the organs’ bile ducts. In this article, Dr Fotios Sampaziotis explains how his team’s study provides the first proof-of-principle for the efficacy of cellular therapies using organoids in human.
Covalent fragment screening in cell-based phenotypic models of disease: a collaborative approach
The application of chemical perturbation approaches in phenotypic models is often used to identify protein targets for therapeutic discovery. Increasingly, small molecule fragments which covalently bind to their protein targets are being used to explore the druggable proteome and the resulting fragment‑protein interactions are characterised by chemoproteomic techniques. In this…