{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,18]],"date-time":"2026-05-18T07:09:58Z","timestamp":1779088198121,"version":"3.51.4"},"reference-count":10,"publisher":"Springer Science and Business Media LLC","issue":"4","license":[{"start":{"date-parts":[[2023,2,27]],"date-time":"2023-02-27T00:00:00Z","timestamp":1677456000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2023,2,27]],"date-time":"2023-02-27T00:00:00Z","timestamp":1677456000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["EMBO Mol Med"],"published-print":{"date-parts":[[2023,4,11]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    Over the last two decades, gene therapy has given hope of potential cure for many rare diseases. In the simplest form, gene therapy is the transfer or editing of a genetic material to cure a disease via nonviral or viral vehicles. Gene therapy can be performed either\n                    <jats:italic>in vivo<\/jats:italic>\n                    by injecting a vector carrying the gene or tools for gene editing directly into a tissue or into the systemic circulation, or\n                    <jats:italic>ex vivo<\/jats:italic>\n                    when patient cells are genetically modified outside of the body and then introduced back into the patient (Yilmaz\n                    <jats:italic>et al<\/jats:italic>\n                    , 2022). Adeno\u2010associated viral vectors (AAV) have been the vectors of choice for\n                    <jats:italic>in vivo<\/jats:italic>\n                    gene therapy. There has been a lot of promising research on the development of novel tissue and cell\u2010specific serotypes in order to improve efficacy and safety for clinical applications (Kuzmin\n                    <jats:italic>et al<\/jats:italic>\n                    , 2021). In this issue of EMBO Molecular Medicine, Boffa and colleagues present a novel AAV\u2010based liver\u2010directed gene therapy for ornithine aminotransferase deficiency.\n                  <\/jats:p>","DOI":"10.15252\/emmm.202217285","type":"journal-article","created":{"date-parts":[[2023,2,27]],"date-time":"2023-02-27T04:58:32Z","timestamp":1677473912000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Targeting the liver to treat the eye"],"prefix":"10.1038","volume":"15","author":[{"given":"Berna","family":"Seker Yilmaz","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9712-6122","authenticated-orcid":false,"given":"Paul","family":"Gissen","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2023,2,27]]},"reference":[{"key":"BFEMMM202217285_CR1","doi-asserted-by":"crossref","first-page":"497","DOI":"10.1007\/s10545-017-0053-3","volume":"40","author":"J Baruteau","year":"2017","unstructured":"Baruteau J, Waddington SN, Alexander IE, Gissen P (2017) Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects. J Inherit Metab Dis 40: 497\u2013517","journal-title":"J Inherit Metab Dis"},{"key":"BFEMMM202217285_CR2","volume":"15","author":"I Boffa","year":"2022","unstructured":"Boffa I, Polishchuk E, De Stefano L, Dell'Aquila F, Nusco E, Marrocco E, Audano M, Pedretti S, Caterino M, Bellezza I et al (2022) Liver\u2010directed gene therapy for ornithine aminotransferase deficiency. EMBO Mol\u00a0Med 15:\u00a0e17033","journal-title":"EMBO Mol\u00a0Med"},{"key":"BFEMMM202217285_CR3","doi-asserted-by":"crossref","DOI":"10.3390\/biology6010018","volume":"6","author":"A Ginguay","year":"2017","unstructured":"Ginguay A, Cynober L, Curis E, Nicolis I (2017) Ornithine aminotransferase, an important glutamate\u2010metabolizing enzyme at the crossroads of multiple metabolic pathways. 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Biochim Biophys Acta Proteins Proteom 1869: 140555","journal-title":"Biochim Biophys Acta Proteins Proteom"},{"key":"BFEMMM202217285_CR8","doi-asserted-by":"crossref","first-page":"879","DOI":"10.1089\/hum.2022.169","volume":"33","author":"AC Nathwani","year":"2022","unstructured":"Nathwani AC, McIntosh J, Sheridan R (2022) Liver gene therapy. Hum Gene Ther 33: 879\u2013888","journal-title":"Hum Gene Ther"},{"key":"BFEMMM202217285_CR9","doi-asserted-by":"crossref","first-page":"849","DOI":"10.1016\/S0140-6736(17)31868-8","volume":"390","author":"S Russell","year":"2017","unstructured":"Russell S, Bennett J, Wellman JA, Chung DC, Yu Z\u2010F, Tillman A, Wittes J, Pappas J, Elci O, McCague S et al (2017) Efficacy and safety of voretigene neparvovec (AAV2\u2010hRPE65v2) in patients with RPE65\u2010mediated inherited retinal dystrophy: a randomised, controlled, open\u2010label, phase 3 trial. 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