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Antisense oligonucleotide therapies for Amyotrophic Lateral Sclerosis: Existing and emerging targets

20 Mar 2019

Abstract excerpt

Amyotrophic lateral sclerosis (ALS) is a disease with highly heterogenous causes, most of which remain unknown, a multitude of possible disease mechanisms, and no therapy currently available that can halt disease progression. However, recent advances in antisense oligonucleotides have made them a viable option for targeted therapeutics for patients. These molecules offer a method of targeting RNA that is highly...

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PMID: 30904737
DOI: 10.1016/j.biocel.2019.03.009

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