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Genome editing based on paired RNA-guided nucleases (RGNs) from CRISPR\/Cas9 systems has been proposed for permanently repairing faulty\n                    <jats:italic>DMD<\/jats:italic>\n                    loci. However, such multiplexing strategies require the development and testing of delivery systems capable of introducing the various gene editing tools into target cells. Here, we investigated the suitability of adenoviral vectors (AdVs) for multiplexed\n                    <jats:italic>DMD<\/jats:italic>\n                    editing by packaging in single vector particles expression units encoding the\n                    <jats:italic>Streptococcus pyogenes<\/jats:italic>\n                    Cas9 nuclease and sequence-specific gRNA pairs. These RGN components were customized to trigger short- and long-range intragenic\n                    <jats:italic>DMD<\/jats:italic>\n                    excisions encompassing reading frame-disrupting exons in patient-derived muscle progenitor cells. By allowing synchronous and stoichiometric expression of the various RGN components, we demonstrate that dual RGN-encoding AdVs can correct over 10% of target\n                    <jats:italic>DMD<\/jats:italic>\n                    alleles, readily leading to the detection of Becker-like dystrophin proteins in unselected muscle cell populations. Moreover, we report that AdV-based gene editing can be tailored for removing mutations located within the over 500-kb major\n                    <jats:italic>DMD<\/jats:italic>\n                    mutational hotspot. Hence, this single\n                    <jats:italic>DMD<\/jats:italic>\n                    editing strategy can in principle tackle a broad\n                    <jats:italic>spectrum<\/jats:italic>\n                    of mutations present in more than 60% of patients with DMD.\n                  <\/jats:p>","DOI":"10.1038\/srep37051","type":"journal-article","created":{"date-parts":[[2016,11,15]],"date-time":"2016-11-15T05:39:18Z","timestamp":1479188358000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":73,"title":["Adenoviral vectors encoding CRISPR\/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells"],"prefix":"10.1038","volume":"6","author":[{"given":"Ignazio","family":"Maggio","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jin","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Josephine M.","family":"Janssen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoyu","family":"Chen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manuel A. 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