Amyotrophic lateral sclerosis (ALS) is a fatal motor disease in adults. Its pathophysiology\nremains mysterious, but tremendous advances have been made with the discovery of the most\nfrequent mutations of its more common familial form linked to the C9ORF72 gene. Although\nmost cases are still considered sporadic, these genetic mutations have revealed the role of RNA\nproduction, processing and transport in ALS, and may be important players in all ALS forms.\nThere are no disease-modifying treatments for adult human neurodegenerative diseases, including\nALS. As in spinal muscular atrophy, RNA-targeted therapies have been proposed as potential\nstrategies for treating this neurodegenerative disorder. Successes achieved in various animal models\nof ALS have proven that RNA therapies are both safe and effective. With careful consideration of the\napplicability of such therapies in humans, it is possible to anticipate ongoing in vivo research and\nclinical trial development of RNA therapies for treating ALS.
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