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Among the many cis-elements involved in the splicing regulation of SMN exon 7, intronic splicing silencer N1 (ISS-N1) has emerged as the most effective target for an antisense oligonucleotide (ASO)-mediated splicing correction of SMN2 exon 7. Blocking of ISS-N1 by an ASO has been shown to fully restore SMN2 exon 7 inclusion in SMA patient cells as well as in vivo. Here we review how ISS-N1 targeting ASOs that use different chemistries respond differently in the various SMA mouse models. We also compare other ASO-based strategies for therapeutic splicing correction in SMA. Given that substantial progress on ASO-based strategies to promote SMN2 exon 7 inclusion in SMA has been made, and that similar approaches in a growing number of genetic diseases are possible, this report has wide implications.<\/jats:p>","DOI":"10.2478\/s13380-013-0109-2","type":"journal-article","created":{"date-parts":[[2013,3,6]],"date-time":"2013-03-06T07:51:17Z","timestamp":1362556277000},"page":"1-7","source":"Crossref","is-referenced-by-count":41,"title":["Antisense oligonucleotide mediated therapy of spinal muscular atrophy"],"prefix":"10.2478","volume":"4","author":[{"given":"Senthilkumar","family":"Sivanesan","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew","family":"Howell","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christine","family":"DiDonato","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ravindra","family":"Singh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"374","reference":[{"key":"109_CR1","doi-asserted-by":"crossref","first-page":"919","DOI":"10.1016\/S0140-6736(80)90847-8","volume":"8174","author":"J Pearn","year":"1980","unstructured":"Pearn J., Classification of spinal muscular atrophies, Lancet, 1980, 8174, 919\u2013922","journal-title":"Lancet"},{"key":"109_CR2","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/0092-8674(95)90460-3","volume":"80","author":"S Lefebvre","year":"1995","unstructured":"Lefebvre S., Burglen L., Reboullet S., Clermont O., Burlet P., Viollet L., et al., Identification and characterization of a spinal muscular atrophydetermining gene, Cell, 1995, 80, 1\u20135","journal-title":"Cell"},{"key":"109_CR3","doi-asserted-by":"crossref","first-page":"1411","DOI":"10.1086\/515465","volume":"60","author":"P E McAndrew","year":"1997","unstructured":"McAndrew P. 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