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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, Duchenne muscular dystrophy and familial amyloid polyneuropathy to highlight the different mechanisms of action of ASOs and RNAi.<\/jats:p>","DOI":"10.1136\/practneurol-2017-001764","type":"journal-article","created":{"date-parts":[[2018,2,17]],"date-time":"2018-02-17T20:20:11Z","timestamp":1518898811000},"page":"126-131","update-policy":"https:\/\/doi.org\/10.1136\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":71,"title":["Antisense oligonucleotides and other genetic therapies made simple"],"prefix":"10.1136","volume":"18","clinical-trial-number":[{"clinical-trial-number":"nct02519036","registry":"10.18810\/clinical-trials-gov"},{"clinical-trial-number":"nct02623699","registry":"10.18810\/clinical-trials-gov"},{"clinical-trial-number":"nct02341560","registry":"10.18810\/clinical-trials-gov"}],"author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4648-2896","authenticated-orcid":false,"given":"Alexander M","family":"Rossor","sequence":"first","affiliation":[{"name":"MRC Centre for Neuromuscular Diseases, UCL Institute of Neurology and the National Hospital for Neurology and Neurosurgery, London, UK"},{"name":"Sobell Department of Motor Neuroscience and Movement Disorders, Institute of Neurology, University College London, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mary M","family":"Reilly","sequence":"additional","affiliation":[{"name":"MRC Centre for Neuromuscular Diseases, UCL Institute of Neurology and the National Hospital for Neurology and Neurosurgery, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James N","family":"Sleigh","sequence":"additional","affiliation":[{"name":"Sobell Department of Motor Neuroscience and Movement Disorders, Institute of Neurology, University College London, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"239","published-online":{"date-parts":[[2018,2,17]]},"reference":[{"key":"2025090409461958000_18.2.126.1","doi-asserted-by":"crossref","first-page":"1056","DOI":"10.1016\/j.neuron.2017.04.010","article-title":"Antisense oligonucleotides: translation from mouse models to human neurodegenerative diseases","volume":"94","author":"Schoch","year":"2017","journal-title":"Neuron"},{"key":"2025090409461958000_18.2.126.2","doi-asserted-by":"crossref","first-page":"60","DOI":"10.1186\/s13073-017-0450-0","article-title":"Advances in the delivery of RNA therapeutics: from concept to clinical reality","volume":"9","author":"Kaczmarek","year":"2017","journal-title":"Genome Med"},{"key":"2025090409461958000_18.2.126.3","doi-asserted-by":"crossref","first-page":"1793","DOI":"10.4155\/fmc.15.101","article-title":"Mechanistic principles of antisense targets for the treatment of spinal muscular atrophy","volume":"7","author":"Singh","year":"2015","journal-title":"Future Med Chem"},{"key":"2025090409461958000_18.2.126.4","doi-asserted-by":"publisher","DOI":"10.1016\/j.addr.2015.05.008"},{"key":"2025090409461958000_18.2.126.5","doi-asserted-by":"publisher","DOI":"10.3109\/13506129.2012.673140"},{"key":"2025090409461958000_18.2.126.6","doi-asserted-by":"publisher","DOI":"10.1038\/bjp.2008.349"},{"key":"2025090409461958000_18.2.126.7","doi-asserted-by":"crossref","first-page":"1713","DOI":"10.1056\/NEJMoa1706198","article-title":"Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy","volume":"377","author":"Mendell","year":"2017","journal-title":"N Engl J Med"},{"key":"2025090409461958000_18.2.126.8","doi-asserted-by":"crossref","first-page":"465","DOI":"10.1038\/mt.2016.5","article-title":"Current progress in therapeutic gene editing for monogenic diseases","volume":"24","author":"Prakash","year":"2016","journal-title":"Mol Ther"},{"key":"2025090409461958000_18.2.126.9","doi-asserted-by":"crossref","first-page":"534","DOI":"10.1038\/gt.2017.32","article-title":"New treatments for serious conditions: ethical implications","volume":"24","author":"King","year":"2017","journal-title":"Gene Ther"},{"key":"2025090409461958000_18.2.126.10","doi-asserted-by":"crossref","unstructured":"Tosolini AP , Sleigh JN . 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