Antisense oligonucleotides for neurodegeneration

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Science  27 Mar 2020:
Vol. 367, Issue 6485, pp. 1428-1429
DOI: 10.1126/science.aba4624


Antisense oligonucleotides (ASOs) have the potential to reduce, restore, or modify RNA and protein expression. Thus, they can target disease pathogenesis by altering the expression of mutant proteins (1). The recent regulatory approval of ASOs for the pediatric motor neuron disease spinal muscular atrophy has provided a regulatory pathway for additional ASO therapies in other central nervous system (CNS) diseases. Developments in ASO chemistry and advances in CNS delivery methods have enabled ASOs to enter clinical trials to treat Huntington's disease (HD). There are currently no available treatments that slow or prevent progression of HD, but two ongoing ASO-based clinical programs have shown promising results. Additionally, clinical trials of ASOs to treat amyotrophic lateral sclerosis (ALS), Parkinson's disease, and Alzheimer's disease are under way, with more in development for other neurodegenerative diseases. It is hoped that ASO-based approaches will provide effective diseasemodifying therapies for HD and similar neurodegenerative diseases soon.

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