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.
In the scientific community there is an emphasis on positive results: getting published, having a drug be approved, etc. However, crucial learning happens when experiments don’t work – in fact, these “failures” may be some of the most important learning experiences. In this article we discuss the need to shift the focus from failing less, to failing faster and some of the resources available to researchers so they can expedite their own work.
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.
Learn more about the DrugAge Database which highlights the high translational value of C. elegans as a model organism, and shows how valuable they can be as a tool for identifying novel drugs and drug targets in longevity research.
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).
With the advent of the molecular biology revolution and the human genome project in the 1990s there was a historic shift toward target-based drug development. Target-based drug development, or TDD, relies on prior knowledge of disease mechanisms and targets before development can begin.
Tune in weekly to our virtual series “Seventeen Minutes of Science” every Tuesday at 11am PST / 2pm ET where we go live on Facebook with a new guest each week to talk about how science and biotechnology is woven into their lives for (you guessed it) 17 minutes! This week on 17 Minutes of …