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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.
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.
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…
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…
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…
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…
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.
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…
A new promising sarcoma target, phosphodiesterase 3A (PDE3A), and drugs targeting it have been identified by researchers at the University of Helsinki. Dr Katja Ivanitskiy, Dr Harri Sihto and Professor Olli Kallioniemi outline emerging evidence that indicates PDE3A protein-targeting compounds may induce sarcoma cell death by acting as a molecular…
With the COVID-19 pandemic ongoing, new therapeutic drugs to combat SARS-CoV-2 are still required. In this article, Professor Arvind Patel from the Medical Research Council (MRC) – Centre for Virus Research (CVR) at the University of Glasgow spoke with Drug Target Review’s Victoria Rees to discuss the work being done…
In this article, Drug Target Review’s Victoria Rees explores a new screening platform that assesses the biological activity of molecules to identify potential drugs. Using their new technique, researchers from the US National Institutes of Health (NIH) identified potential COVID-19 therapeutics.
A major challenge during high-throughput and fragment screening is the potential for identifying ‘frequent hitters’ – compounds that affect unrelated targets. Matthew Lloyd from the University of Bath explains why these hits can arise during drug discovery and how machine learning could be the answer to identifying these compounds.
Optimisation of a hit towards a lead happens in a variety of ways, as a lead needs to meet various criteria such as activity, affinity to the target, selectivity, solubility, permeability or metabolic stability to become a promising candidate. A balance between in vitro profiles and pharmacokinetic attributes has to…
While researchers conduct their studies, constraints such as time can impact their work. Dr Ian Holland from the University of Edinburgh spoke with Drug Target Review’s Deputy Editor Victoria Rees to explain how lab automation can offer a solution to these challenges and enhance output for scientists.
Advances in genomic medicine are playing an increasingly important role in the field of cardiology. Better analysis and understanding of patient genomic and epigenomic information can enable more personalised patient treatment and medical intervention. Here, Professor John Giannios considers the potential to use genomic medicine to prevent, monitor, diagnose and…
