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We describe genome editing of\n                    <jats:italic>SMN2<\/jats:italic>\n                    , an insufficient copy of\n                    <jats:italic>SMN1<\/jats:italic>\n                    harboring a C6&gt;T mutation, to permanently restore SMN protein levels and rescue SMA phenotypes. We used nucleases or base editors to modify five\n                    <jats:italic>SMN2<\/jats:italic>\n                    regulatory regions. Base editing converted\n                    <jats:italic>SMN2<\/jats:italic>\n                    T6&gt;C, restoring SMN protein levels to wild type. Adeno-associated virus serotype 9\u2013mediated base editor delivery in \u03947SMA mice yielded 87% average T6&gt;C conversion, improved motor function, and extended average life span, which was enhanced by one-time base editor and nusinersen coadministration (111 versus 17 days untreated). These findings demonstrate the potential of a one-time base editing treatment for SMA.\n                  <\/jats:p>","DOI":"10.1126\/science.adg6518","type":"journal-article","created":{"date-parts":[[2023,3,30]],"date-time":"2023-03-30T14:00:09Z","timestamp":1680184809000},"update-policy":"https:\/\/doi.org\/10.34133\/aaas_crossmark","source":"Crossref","is-referenced-by-count":140,"title":["Base editing rescue of spinal muscular atrophy in cells and in mice"],"prefix":"10.1126","volume":"380","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9216-5096","authenticated-orcid":true,"given":"Mandana","family":"Arbab","sequence":"first","affiliation":[{"name":"Department of Neurology, Rosamund Stone Zander Translational Neuroscience Center, Boston Children\u2019s Hospital, Boston, MA 02115, USA."},{"name":"Department of Neurobiology, Harvard Medical School, Boston, MA 02115, USA."},{"name":"Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and 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