About this Research Topic
Our goal is to present and evaluate the current state of neuromuscular disease models for the purposes of uncovering molecular mechanisms and for drug discovery. We will highlight recent advances in iPSC directed differentations to lineages encompassed by neuromuscular diseases—namely, skeletal muscle as well as motor and sensory neurons. We will discuss how closely human stem cell-based models recapitulate neuromuscular disease etiology and progression. Furthermore, we will review when and why stem cell models align with well accepted animal models and when and how they don’t. Advances in gene editing technology has contributed a great deal to this endeavor for both modeling and potential therapeutic interventions. The strengths and weaknesses of this technology in both domains will be covered. What do the fields of bioengineering and materials science contribute to these disease models in terms of recreating the native microenvironment?
1. How to define a good disease model?
2. Comparison of animal models to stem cell-based ‘disease-in-a-dish’ models; strengths and weaknesses of both
3. Does the disease model recapitulate symptoms observed in the patients and are those manifestations rooted in evolutionarily conserved biochemistry/molecular biology?
4. Have iPSC disease models reached a level of sophistication that they can substitute for animal models?
5. If so, what impact with they have on the rather dismal success rates of clinical trials and how soon will that impact be reflected in decreasing cost, time to market and/or correct predictions of efficacy and safety?
6. When is it necessary to switch from a 2D disease model to a 3D model to achieve the necessary level of clinical predictive power? Is the additional complexity of a 3D model worth the time and cost?
7. What do other disciplines bring to disease modeling efforts? Bioengineering, biomaterials, etc
Topic Editor David Mack is a founder of KineaBio Inc. Topic Editor Megan Laura McCain hold a patents related to the theme of the collection.
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