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The Application of Hypersensitivity Assays to the Discovery of Therapeutics

The Application of Hypersensitivity Assays to the Discovery of Therapeutics

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.

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Let’s Fail Faster: Why Scientific Failures Should be Accepted as Progress

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.

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The Path to Affordable Therapeutics in Rare Disease – Tackling Congenital Disorder of Glycosylation in the PMM2 gene.

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.

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Seventeen Minutes of Science: Viral Gene Therapies and Their Potential Use Against COVID-19

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 …

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