{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,17]],"date-time":"2026-08-17T14:15:41Z","timestamp":1786976141070,"version":"build-2736575974"},"reference-count":138,"publisher":"Wiley","license":[{"start":{"date-parts":[[2024,7,30]],"date-time":"2024-07-30T00:00:00Z","timestamp":1722297600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100011033","name":"Agencia Estatal de Investigaci\u00f3n","doi-asserted-by":"publisher","award":["PID2021\u2010124455OB\u2010I00"],"award-info":[{"award-number":["PID2021\u2010124455OB\u2010I00"]}],"id":[{"id":"10.13039\/501100011033","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100011033","name":"Agencia Estatal de Investigaci\u00f3n","doi-asserted-by":"publisher","award":["RTI2018\u2010101936\u2010B\u2010I00"],"award-info":[{"award-number":["RTI2018\u2010101936\u2010B\u2010I00"]}],"id":[{"id":"10.13039\/501100011033","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004587","name":"Instituto de Salud Carlos III","doi-asserted-by":"publisher","award":["TERAVRD21\/0017\/0001"],"award-info":[{"award-number":["TERAVRD21\/0017\/0001"]}],"id":[{"id":"10.13039\/501100004587","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["FEBS Letters"],"abstract":"<jats:p>\n                    The deliberate and precise modification of the host genome using engineered nucleases represents a groundbreaking advancement in modern medicine. Several clinical trials employing these approaches to address metabolic liver disorders have been initiated, with recent remarkable outcomes observed in patients with transthyretin amyloidosis, highlighting the potential of these therapies. Recent technological improvements, particularly CRISPR Cas9\u2010based technology, have revolutionized gene editing, enabling\n                    <jats:italic>in vivo<\/jats:italic>\n                    modification of the cellular genome for therapeutic purposes. These modifications include gene supplementation, correction, or silencing, offering a wide range of therapeutic possibilities. Moving forward, we anticipate witnessing the unfolding therapeutic potential of these strategies in the coming years. The aim of our review is to summarize preclinical data on gene editing in animal models of inherited liver diseases and the clinical data obtained thus far, emphasizing both therapeutic efficacy and potential limitations of these medical interventions.\n                  <\/jats:p>","DOI":"10.1002\/1873-3468.14989","type":"journal-article","created":{"date-parts":[[2024,7,30]],"date-time":"2024-07-30T22:45:01Z","timestamp":1722379501000},"source":"Crossref","is-referenced-by-count":5,"title":["Gene editing in liver diseases"],"prefix":"10.1002","author":[{"given":"Laura","family":"Torella","sequence":"first","affiliation":[{"name":"DNA &amp; RNA Medicine Division, Gene Therapy for Rare Diseases Department, Center for Applied Medical Research (CIMA) University of Navarra, IdisNA  Pamplona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nerea","family":"Santana\u2010Gonzalez","sequence":"additional","affiliation":[{"name":"DNA &amp; RNA Medicine Division, Gene Therapy for Rare Diseases Department, Center for Applied Medical Research (CIMA) University of Navarra, IdisNA  Pamplona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nerea","family":"Zabaleta","sequence":"additional","affiliation":[{"name":"Grousbeck Gene Therapy Center Schepens Eye Research Institute, Mass Eye and Ear  Boston MA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1600-4562","authenticated-orcid":false,"given":"Gloria","family":"Gonzalez Aseguinolaza","sequence":"additional","affiliation":[{"name":"DNA &amp; RNA Medicine Division, Gene Therapy for Rare Diseases Department, Center for Applied Medical Research (CIMA) University of Navarra, IdisNA  Pamplona Spain"},{"name":"Vivet Therapeutics  Pamplona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,7,30]]},"reference":[{"key":"e_1_2_11_2_1","first-page":"30150","article-title":"Clinical and biochemical footprints of inherited metabolic diseases. 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