{"id":3733,"date":"2021-01-19T15:11:59","date_gmt":"2021-01-19T20:11:59","guid":{"rendered":"https:\/\/www.med.unc.edu\/bloodresearchcenter\/?p=3733"},"modified":"2021-01-19T15:15:14","modified_gmt":"2021-01-19T20:15:14","slug":"nature-biotechnology-paper-published-timothy-nichols-md-is-one-of-the-authors","status":"publish","type":"post","link":"https:\/\/www.med.unc.edu\/bloodresearchcenter\/2021\/01\/nature-biotechnology-paper-published-timothy-nichols-md-is-one-of-the-authors\/","title":{"rendered":"Nature Biotechnology paper published, Timothy Nichols, MD"},"content":{"rendered":"<h4><em>A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells<\/em>.<\/h4>\n<p>Authors: Giang N. Nguyen, John K. Everett, Samita Kafle, Aoife M. Roche, Hayley E. Raymond, Jacob Leiby, Christian Wood, Charles-Antoine Assenmacher, Elizabeth P. Merricks, C. Tyler Long, Haig H. Kazazian, Timothy C. Nichols, Frederic D. Bushman and Denise E. Sabatino.<\/p>\n<p>Dogs with hemophilia A due to an intron 22 inversion were treated with AAV vectors driving expression of canine FVIII and followed for over 11 years.\u00a0 Canine FVIII expression persisted in all dogs and increased in at least 2.\u00a0 This is the longest duration of expression reported to date in any animal model.\u00a0 Vector integration studies revealed genomic integration mostly of vector fragments but in at least on case intact vector.\u00a0 Vector integration occurred at or near multiple genes including transcription factors \u00a0in the canine genome. Clonal expansion was also observed.\u00a0 Despite these findings of vector integration and clonal expansion,\u00a0 no hepatocellular carcinomas were found.\u00a0 Future work is planned to determine whether or not AAV vector integration is causing clonal expansion. \u00a0The data strongly support the safety of current AAV vectors but also highlight areas for potential improvements and optimization.<\/p>\n<p><strong><a href=\"https:\/\/www.med.unc.edu\/bloodresearchcenter\/wp-content\/uploads\/sites\/1089\/2021\/01\/NguyenEverett2020.pdf\">Read full article here<\/a><\/strong><\/p>\n","protected":false},"excerpt":{"rendered":"<p>A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells. Authors: Giang N. Nguyen, John K. Everett, Samita Kafle, Aoife M. Roche, Hayley E. Raymond, Jacob Leiby, Christian Wood, Charles-Antoine Assenmacher, Elizabeth P. Merricks, C. Tyler Long, Haig H. Kazazian, Timothy C. Nichols, Frederic D. Bushman &hellip; <a href=\"https:\/\/www.med.unc.edu\/bloodresearchcenter\/2021\/01\/nature-biotechnology-paper-published-timothy-nichols-md-is-one-of-the-authors\/\" aria-label=\"Read more about Nature Biotechnology paper published, Timothy Nichols, MD\">Read more<\/a><\/p>\n","protected":false},"author":15383,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"layout":"","cellInformation":"","apiCallInformation":"","footnotes":"","_links_to":"","_links_to_target":""},"categories":[2],"tags":[],"class_list":["post-3733","post","type-post","status-publish","format-standard","hentry","category-news","odd"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v26.8 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Nature Biotechnology paper published, Timothy Nichols, MD | UNC Blood Research Center<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.med.unc.edu\/bloodresearchcenter\/2021\/01\/nature-biotechnology-paper-published-timothy-nichols-md-is-one-of-the-authors\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Nature Biotechnology paper published, Timothy Nichols, MD | UNC Blood Research Center\" \/>\n<meta property=\"og:description\" content=\"A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells. Authors: Giang N. Nguyen, John K. Everett, Samita Kafle, Aoife M. Roche, Hayley E. Raymond, Jacob Leiby, Christian Wood, Charles-Antoine Assenmacher, Elizabeth P. Merricks, C. Tyler Long, Haig H. Kazazian, Timothy C. Nichols, Frederic D. 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