Using Humanization we can take advantage of the ancient biology between humans and other organisms to create stand-ins – patient avatars – for drug screening studies. In this blog, we will focus on models of inborn errors of metabolism (IEM), as these genetic conditions can lead to hypersensitivity to the metabolite. Since stressor condition hypersensitivity can be used to detect favorable drug effects, IEM model systems are ideal tools for phenotypic screens to find molecules that alleviate the metabolic stress occurring from the deficiency. We discuss the model organisms used in hypersensitivity screens, and why they are advantageous to drug discovery. Ultimately, showcasing this approach’s potential to be widely generalized to many genetic disorders.
Reactive oxygen species (ROS) are free radicals that have been found to extend lifespan at low levels, however, they are lethal at higher doses. So is the saying ‘what doesn’t kill you make you stronger’ true? Or should free radicals be avoided as much as possible? In this article we discuss the current paradoxical findings, and focus on genetic disorders such as Progeria which is characterized by rapid aging and elevated ROS levels.
Rare diseases are just that: rare. However, when combined rare diseases affect 3.5% – 5.9% of the worldwide population, and present a growing healthcare issue. In this article we talk with Dr Sangetha lyer about her work at the biotech Perlara and highlight their recent successes in a drug repurposing campaign for PMM2-CDG.
We have found whole gene humanization can create a platform for highly translatable results in a model organism. Currently there is a big need for functional studies of Variants of Uncertain Significance (VUS).
Using zebrafish modeling in a rare human disease may be the perfect physiologic model to better understand the disease and generate more individualized therapeutic medical responses and positive outcomes for higher risk COVID-19 groups.
NemaMetrix is the market leader for early preclinical in-vivo testing using small animal models to gain a better understanding of the efficacy, mode of action, toxicity and potential targets for novel compounds. In this case study we outline how we used the animal model C. elegans to perform a set of specific experiments designed by our experts to provide rapid and reliable data based on our customer’s priorities. Using the data that we provided, our customer was able to apply for multiple patents in a timely manner.