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Antisense oligonucleotides and other genetic therapies made simple

17 Feb 2018

Abstract excerpt

Many genetic neurological diseases result from the dysfunction of single proteins. Genetic therapies aim to modify these disease-associated proteins by targeting the RNA and DNA precursors. This review provides a brief overview of the main types of genetic therapies, with a focus on antisense oligonucleotides (ASOs) and RNA interference (RNAi). We use examples of new genetic therapies for spinal muscular atrophy,...

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PMID: 29455156
DOI: 10.1136/practneurol-2017-001764

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