"Amyotrophic Lateral Sclerosis: Recent Advances and Therapeutic Challenges" ed. by Muralidhar L. Hegde

This book highlights emerging science in the area of Amyotrophic Lateral Sclerosis (ALS) and discusses key approaches and mechanisms essential for developing a cure for ALS.

A flurry of recent research on the role of the RNA/DNA-binding proteins TDP-43 and FUS as well as a dozen other factors (e.g., C9ORF72 and profilin) has led to a new paradigm in our understanding of the pathobiology of the motor neuron disease, Amyotrophic Lateral Sclerosis (ALS). How these factors trigger neuromuscular dysfunction is critical for developing more effective ALS therapeutics. The ‘gain-of-toxicity’ or ‘loss-of-function’ of these etiological factors is a key question. Recent studies on the imbalance in genome damage versus repair have opened avenues for potential DNA repair-based therapeutics.

Contents
1.Molecular Basis of DNA Repair Defects in FUS-Associated ALS: Implications of a New Paradigm and Its Potential as Therapeutic Target
2.Senataxin: A Putative RNA: DNA Helicase Mutated in ALS4—Emerging Mechanisms of Genome Stability in Motor Neurons
3.Pathological Interaction between DNA Repair and Mitochondrial Dysfunction in ALS
4.The Role of TDP-43 in Genome Repair and beyond in Amyotrophic Lateral Sclerosis
5.The Extraocular Muscles Are Selectively Spared in ALS
6.The Role of Extracellular Vesicles in the Progression of ALS and Their Potential as Biomarkers and Therapeutic Agents with Which to Combat the Disease
7.RNA Metabolism and Therapeutics in Amyotrophic Lateral Sclerosis
8.Targeting Axonal Transport: A New Therapeutic Avenue for ALS