Research Topic

RNA toxicity mechanisms and therapeutic development in neurological and neuromuscular disorders

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About this Research Topic

A group of late-onset, progressive neurodegenerative/neuromuscular disorders are caused by the abnormal nucleotide expansion mechanism in disease genes. They include Fragile-X Syndrome, Friedreich ataxia, Frontotemporal dementia/Amyotrophic lateral sclerosis, Huntington's Disease, Myotonic Dystrophy (DM) and ...

A group of late-onset, progressive neurodegenerative/neuromuscular disorders are caused by the abnormal nucleotide expansion mechanism in disease genes. They include Fragile-X Syndrome, Friedreich ataxia, Frontotemporal dementia/Amyotrophic lateral sclerosis, Huntington's Disease, Myotonic Dystrophy (DM) and Spinocerebellar Ataxias (SCAs). The recent rapid advances in the mechanistic studies and therapeutic development of this group of diseases raise the possibility of novel treatment strategies. Although diverse toxic pathways have been identified in these disorders which help explain the molecular pathogenesis of individual disorders, new ones are reported every once in a while. Even an already described pathogenic event may further been shown to affect additional diseases. This highlights the sharing of toxic pathways among this group of “originally-thought-to-be diverse disorders”. For instance, the involvement of Repeat-associated non-ATG translation (RAN translation) was first described in spinocerebellar ataxia type 8 and DM1, but has more recently been described in patients with the C9orf72 mutation. Due to the sharing of common pathogenic pathways, it is reasonable to speculate that therapeutic strategies that were once proved to be effective for neutralizing one disease may also be a viable treatment direction for other disorders. A Research Topic in this area will surely help consolidate both the recent findings (review articles) and frontier advances (research articles) in the pathogenesis and therapeutic treatments for repeat expansion-related neurodegenerative/neuromuscular disorders.


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