Current opinion in neurology
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Curr. Opin. Neurol. · Oct 2018
ReviewEmerging antisense oligonucleotide and viral therapies for amyotrophic lateral sclerosis.
Amyotrophic lateral sclerosis (ALS) is a rapidly fatal disease for which there is currently no effective therapy. The present review describes the current progress of existing molecular therapies in the clinical trial pipeline and highlights promising future antisense oligonucleotide (ASO) and viral therapeutic strategies for treating ALS. ⋯ Advances in preclinical and clinical studies of ASO and viral directed approaches to neuromuscular disease, particularly ALS, indicate that these approaches have high specificity and are relatively well tolerated.