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Successful Use of Out-of-Frame Exon 2 Skipping Induces IRES-Driven Expression of the N-Truncated Dystrophin Isoform: Promising Approach for Treating Other 5 ' Dystrophin Mutations
KTH, School of Biotechnology (BIO), Proteomics and Nanobiotechnology.ORCID iD: 0000-0001-6990-1905
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2014 (English)In: Molecular Therapy, ISSN 1525-0016, E-ISSN 1525-0024, Vol. 22, S294-S295 p.Article in journal, Meeting abstract (Other academic) Published
Place, publisher, year, edition, pages
2014. Vol. 22, S294-S295 p.
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Biological Sciences
URN: urn:nbn:se:kth:diva-148261ISI: 000337231300753OAI: diva2:737657
17th Annual Meeting of the American-Society-of-Gene-and-Cell-Therapy (ASGCT), MAY 21-24, 2014, Washington, DC

QC 20140813

Available from: 2014-08-13 Created: 2014-08-04 Last updated: 2014-08-13Bibliographically approved

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Szigyarto, Cristina Al-KhaliliUhlén, Mathias
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