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In utero editing was associated with sustained postnatal CFTR activity, at a level similar to that of wild-type mice, in both respiratory and gastrointestinal tissues, without detection of off-target mutations in partially homologous loci. This work suggests that systemic in utero gene editing represents a viable strategy for treating monogenic diseases before birth that impact multiple tissue types.\n                  <\/jats:p>","DOI":"10.1073\/pnas.2418731122","type":"journal-article","created":{"date-parts":[[2025,6,10]],"date-time":"2025-06-10T11:07:53Z","timestamp":1749553673000},"update-policy":"https:\/\/doi.org\/10.1073\/pnas.cm10313","source":"Crossref","is-referenced-by-count":9,"title":["Systemic in utero gene editing as a treatment for cystic fibrosis"],"prefix":"10.1073","volume":"122","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2637-8522","authenticated-orcid":false,"given":"Adele S.","family":"Ricciardi","sequence":"first","affiliation":[{"name":"Department of Biomedical Engineering, Yale University","place":["New Haven"]},{"name":"Department of Therapeutic Radiology, Yale University","place":["New Haven"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christina","family":"Barone","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Yale University","place":["New Haven"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1953-8452","authenticated-orcid":false,"given":"Rachael","family":"Putman","sequence":"additional","affiliation":[{"name":"Department of Biomedical Engineering, Yale University","place":["New Haven"]},{"name":"Department of Therapeutic Radiology, Yale University","place":["New Haven"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elias","family":"Quijano","sequence":"additional","affiliation":[{"name":"Department of Genetics, Yale University","place":["New Haven"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anisha","family":"Gupta","sequence":"additional","affiliation":[{"name":"Department of Therapeutic Radiology, Yale University","place":["New Haven"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard","family":"Nguyen","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Yale University","place":["New Haven"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hanna","family":"Mandl","sequence":"additional","affiliation":[{"name":"Department of Biomedical Engineering, Yale University","place":["New Haven"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4555-3234","authenticated-orcid":false,"given":"Alexandra S.","family":"Piotrowski-Daspit","sequence":"additional","affiliation":[{"name":"Department of Biomedical Engineering, Yale University","place":["New Haven"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7476-9822","authenticated-orcid":false,"given":"Francesc","family":"Lopez-Giraldez","sequence":"additional","affiliation":[{"name":"Department of Genetics, Yale University","place":["New Haven"]},{"name":"Yale Center for Genome Analysis","place":["New Haven"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Valerie","family":"Luks","sequence":"additional","affiliation":[{"name":"Department of Surgery, Yale University","place":["New Haven"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mollie R.","family":"Freedman-Weiss","sequence":"additional","affiliation":[{"name":"Department of Surgery, Yale University","place":["New Haven"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8553-7162","authenticated-orcid":false,"given":"James","family":"Farrelly","sequence":"additional","affiliation":[{"name":"Department of Surgery, Yale University","place":["New Haven"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Samantha","family":"Ahle","sequence":"additional","affiliation":[{"name":"Department of Surgery, Yale University","place":["New Haven"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2659-3284","authenticated-orcid":false,"given":"Anna Y.","family":"Lynn","sequence":"additional","affiliation":[{"name":"Department of Biomedical Engineering, Yale University","place":["New Haven"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4525-5560","authenticated-orcid":false,"given":"Peter M.","family":"Glazer","sequence":"additional","affiliation":[{"name":"Department of Therapeutic Radiology, Yale University","place":["New Haven"]},{"name":"Department of Genetics, Yale University","place":["New Haven"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2163-549X","authenticated-orcid":false,"given":"W. 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