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While current treatment strategies, including small molecules or viral vectors, have shown promise in improving motor function and survival, achieving a definitive and long-term correction of SMA\u2019s endogenous mutations and phenotypes remains highly challenging. We have previously developed a CRISPR-Cas9 based homology-independent targeted integration (HITI) strategy, enabling unidirectional DNA knock-in in both dividing and non-dividing cells in vivo. In this study, we demonstrated its utility by correcting an SMA mutation in mice. When combined with\n                    <jats:italic>Smn1<\/jats:italic>\n                    cDNA supplementation, it exhibited long-term therapeutic benefits in SMA mice. Our observations may provide new avenues for the long-term and efficient treatment of inherited diseases.\n                  <\/jats:p>","DOI":"10.1038\/s41467-024-50095-5","type":"journal-article","created":{"date-parts":[[2024,7,24]],"date-time":"2024-07-24T06:02:09Z","timestamp":1721800929000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":23,"title":["Therapeutic strategy for spinal muscular atrophy by combining gene supplementation and genome editing"],"prefix":"10.1038","volume":"15","author":[{"given":"Fumiyuki","family":"Hatanaka","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5603-9105","authenticated-orcid":false,"given":"Keiichiro","family":"Suzuki","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2852-5226","authenticated-orcid":false,"given":"Kensaku","family":"Shojima","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0005-1182-3680","authenticated-orcid":false,"given":"Jingting","family":"Yu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuta","family":"Takahashi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Akihisa","family":"Sakamoto","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Javier","family":"Prieto","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maxim","family":"Shokhirev","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0554-2365","authenticated-orcid":false,"given":"Estrella","family":"Nu\u00f1ez Delicado","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Concepcion","family":"Rodriguez Esteban","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0557-8875","authenticated-orcid":false,"given":"Juan Carlos","family":"Izpisua Belmonte","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2024,7,24]]},"reference":[{"key":"50095_CR1","doi-asserted-by":"publisher","first-page":"3877","DOI":"10.1007\/s00018-018-2849-1","volume":"75","author":"H Chaytow","year":"2018","unstructured":"Chaytow, H., Huang, Y.-T., Gillingwater, T. 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