{"id":18173,"date":"2025-02-13T20:13:34","date_gmt":"2025-02-13T20:13:34","guid":{"rendered":"https:\/\/www.tun.com\/home\/?p=18173"},"modified":"2025-02-13T20:13:35","modified_gmt":"2025-02-13T20:13:35","slug":"new-gene-editing-method-could-transform-liver-disease-treatment","status":"publish","type":"post","link":"https:\/\/www.tun.com\/home\/new-gene-editing-method-could-transform-liver-disease-treatment\/","title":{"rendered":"New Gene-Editing Method Could Transform Liver Disease Treatment"},"content":{"rendered":"\n<div class=\"wp-block-group\"><div class=\"wp-block-group__inner-container is-layout-constrained wp-block-group-is-layout-constrained\">\n<div class=\"wp-block-uagb-blockquote uagb-block-e7eb3fc3 uagb-blockquote__skin-border uagb-blockquote__stack-img-none\"><blockquote class=\"uagb-blockquote\"><div class=\"uagb-blockquote__content\">Scientists at Rice University and Baylor College of Medicine have created a new gene-editing method that dramatically increases the effectiveness of liver therapies. This technology, which boosts repaired cell rates from 1% to 25%, holds the potential to treat over 700 genetic liver disorders.<\/div><footer><div class=\"uagb-blockquote__author-wrap uagb-blockquote__author-at-left\"><\/div><\/footer><\/blockquote><\/div>\n\n\n\n<div class=\"wp-block-group is-content-justification-space-between is-nowrap is-layout-flex wp-container-core-group-is-layout-b0ffac9c wp-block-group-is-layout-flex\"><div style=\"font-size:16px\" class=\"has-text-align-left wp-block-post-author\"><div class=\"wp-block-post-author__content\"><p class=\"wp-block-post-author__name\">The University Network<\/p><\/div><\/div>\n\n\n<div class=\"wp-block-uagb-social-share uagb-social-share__outer-wrap uagb-social-share__layout-horizontal uagb-block-ee584a31\">\n<div class=\"wp-block-uagb-social-share-child uagb-ss-repeater uagb-ss__wrapper uagb-block-ec619ce7\"><span class=\"uagb-ss__link\" data-href=\"https:\/\/www.facebook.com\/sharer.php?u=\" tabindex=\"0\" role=\"button\" aria-label=\"facebook\"><span class=\"uagb-ss__source-wrap\"><span class=\"uagb-ss__source-icon\"><svg xmlns=\"https:\/\/www.w3.org\/2000\/svg\" viewBox=\"0 0 512 512\"><path d=\"M504 256C504 119 393 8 256 8S8 119 8 256c0 123.8 90.69 226.4 209.3 245V327.7h-63V256h63v-54.64c0-62.15 37-96.48 93.67-96.48 27.14 0 55.52 4.84 55.52 4.84v61h-31.28c-30.8 0-40.41 19.12-40.41 38.73V256h68.78l-11 71.69h-57.78V501C413.3 482.4 504 379.8 504 256z\"><\/path><\/svg><\/span><\/span><\/span><\/div>\n\n\n\n<div class=\"wp-block-uagb-social-share-child uagb-ss-repeater uagb-ss__wrapper uagb-block-32d99934\"><span class=\"uagb-ss__link\" data-href=\"https:\/\/twitter.com\/share?url=\" tabindex=\"0\" role=\"button\" aria-label=\"twitter\"><span class=\"uagb-ss__source-wrap\"><span class=\"uagb-ss__source-icon\"><svg xmlns=\"https:\/\/www.w3.org\/2000\/svg\" viewBox=\"0 0 512 512\"><path d=\"M389.2 48h70.6L305.6 224.2 487 464H345L233.7 318.6 106.5 464H35.8L200.7 275.5 26.8 48H172.4L272.9 180.9 389.2 48zM364.4 421.8h39.1L151.1 88h-42L364.4 421.8z\"><\/path><\/svg><\/span><\/span><\/span><\/div>\n\n\n\n<div class=\"wp-block-uagb-social-share-child uagb-ss-repeater uagb-ss__wrapper uagb-block-1d136f14\"><span class=\"uagb-ss__link\" data-href=\"https:\/\/www.linkedin.com\/shareArticle?url=\" tabindex=\"0\" role=\"button\" aria-label=\"linkedin\"><span class=\"uagb-ss__source-wrap\"><span class=\"uagb-ss__source-icon\"><svg xmlns=\"https:\/\/www.w3.org\/2000\/svg\" viewBox=\"0 0 448 512\"><path d=\"M416 32H31.9C14.3 32 0 46.5 0 64.3v383.4C0 465.5 14.3 480 31.9 480H416c17.6 0 32-14.5 32-32.3V64.3c0-17.8-14.4-32.3-32-32.3zM135.4 416H69V202.2h66.5V416zm-33.2-243c-21.3 0-38.5-17.3-38.5-38.5S80.9 96 102.2 96c21.2 0 38.5 17.3 38.5 38.5 0 21.3-17.2 38.5-38.5 38.5zm282.1 243h-66.4V312c0-24.8-.5-56.7-34.5-56.7-34.6 0-39.9 27-39.9 54.9V416h-66.4V202.2h63.7v29.2h.9c8.9-16.8 30.6-34.5 62.9-34.5 67.2 0 79.7 44.3 79.7 101.9V416z\"><\/path><\/svg><\/span><\/span><\/span><\/div>\n<\/div>\n<\/div>\n<\/div><\/div>\n\n\n\n<p class=\"wp-block-paragraph\">Researchers from Rice University and Baylor College of Medicine (BCM) have developed a new gene-editing strategy called Repair Drive, which significantly enhances the effectiveness of gene therapies in the liver. This advancement, described in a paper <a href=\"https:\/\/www.science.org\/doi\/10.1126\/scitranslmed.adk3920\" target=\"_blank\" rel=\"noopener\" title=\"\">published<\/a> in Science Translational Medicine, could pave the way for treatments addressing around 700 genetic disorders affecting the liver and potentially other organs and tissues.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Gene-editing therapies exist but are often hampered by high costs and the practice of breaking or inactivating defective genes rather than repairing them. <\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Repair Drive changes this narrative by repairing a higher percentage of liver cells \u2014 known as hepatocytes \u2014 effectively giving them an advantage to outcompete unedited and incorrectly edited cells.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">&#8220;For example, homology directed repair is the preferred pathway for fixing genes, but it is only active in the roughly 1% of liver cells that are actively dividing. This limitation has made it nearly impossible to correct genetic mutations in a significant portion of the liver. Our approach is to take that small percentage of precisely repaired cells and give them a reason to divide so that they can replace the unhealthy liver cells,&#8221; co-senior author William Lagor, a professor of integrative physiology at BCM, said in a <a href=\"https:\/\/news.rice.edu\/news\/2025\/rice-bcm-research-achieves-gene-editing-breakthrough-could-improve-treatment-liver\" target=\"_blank\" rel=\"noopener\" title=\"\">news release<\/a>.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">This new technique employs small interfering RNA (siRNA) to temporarily inhibit an essential gene, FAH, crucial for hepatocyte survival. <\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The researchers then introduced a modified version of FAH along with a therapeutic gene, allowing only gene-edited cells to survive and proliferate.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cThis is like giving gene-edited cells a head start in a race,\u201d added co-senior author Gang Bao, the  A.J. Foyt Family Professor of Bioengineering at Rice.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bao\u2019s lab, which has been significantly advancing gene-editing research, conducted next-generation sequencing and bioinformatics analysis to ensure the precision of the gene edits performed with the new Repair Drive platform.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bao highlighted the project&#8217;s collaborative nature and praised the key contributors. He lauded Marco De Giorgi, an assistant professor in the Lagor lab at BCM and the paper&#8217;s first author, for his \u201cpersistence and vision to overcome difficult biological and technical challenges.\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The significance of this research lies in its broader implications for genetic therapies. <\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cWe\u2019re not just focusing on one disease but instead offering a solution that could be applied to a broad range of conditions caused by genetic mutations in the liver,\u201d Bao added.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The potential impact of Repair Drive is immense, offering a new avenue for restoring healthy liver cells and expanding the scope of treatable genetic disorders. <\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Researchers from Rice University and Baylor College of Medicine (BCM) have developed a new gene-editing strategy called Repair Drive, which significantly enhances the effectiveness of gene therapies in the liver. This advancement, described in a paper published in Science Translational Medicine, could pave the way for treatments addressing around 700 genetic disorders affecting the liver [&hellip;]<\/p>\n","protected":false},"author":3,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"single-no-separators","format":"standard","meta":{"_acf_changed":false,"_uag_custom_page_level_css":"","_monsterinsights_skip_tracking":false,"footnotes":""},"categories":[25],"tags":[],"class_list":["post-18173","post","type-post","status-publish","format-standard","hentry","category-science"],"acf":[],"aioseo_notices":[],"aioseo_head":"\n\t\t<!-- All in One SEO 4.9.8 - aioseo.com -->\n\t<meta name=\"description\" content=\"Researchers have developed a gene-editing technology that significantly improves liver cell repair, offering hope for treatment of various genetic disorders.\" \/>\n\t<meta name=\"robots\" content=\"max-image-preview:large\" \/>\n\t<meta name=\"author\" content=\"The University Network\"\/>\n\t<link rel=\"canonical\" href=\"https:\/\/www.tun.com\/home\/new-gene-editing-method-could-transform-liver-disease-treatment\/\" \/>\n\t<meta name=\"generator\" content=\"All in One SEO (AIOSEO) 4.9.8\" \/>\n\t\t<meta property=\"og:locale\" content=\"en_US\" \/>\n\t\t<meta property=\"og:site_name\" content=\"TUN - 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This advancement, described in a paper published in Science Translational Medicine, could pave the way for treatments addressing around 700 genetic disorders affecting the liver&hellip;","_links":{"self":[{"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/posts\/18173","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/comments?post=18173"}],"version-history":[{"count":14,"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/posts\/18173\/revisions"}],"predecessor-version":[{"id":18240,"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/posts\/18173\/revisions\/18240"}],"wp:attachment":[{"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/media?parent=18173"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/categories?post=18173"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/tags?post=18173"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}