Advances in pharmacotherapy for amyotrophic lateral sclerosis

Abstract:

Amyotrophic lateral sclerosis（ALS） is a progressive neurodegenerative disease characterized by the selective loss of upper and lower motor neurons. Effective cures for this disease have not been available yet. However，promising options for the treatment of ALS have been approaching thanks to the growing understanding of ALS pathogenesis and genetics as well as the innovation of gene regulation strategies. This article provides a comprehensive review of advances on antisense oligonucleotide therapy， adeno-associated virus-mediated gene therapy，and cell therapy in ALS.