{"id":219742,"date":"2025-08-09T08:03:55","date_gmt":"2025-08-09T13:03:55","guid":{"rendered":"https:\/\/lifeboat.com\/blog\/2025\/08\/a-boost-for-the-precision-of-genome-editing"},"modified":"2025-08-09T08:03:55","modified_gmt":"2025-08-09T13:03:55","slug":"a-boost-for-the-precision-of-genome-editing","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2025\/08\/a-boost-for-the-precision-of-genome-editing","title":{"rendered":"A Boost for the Precision of Genome Editing"},"content":{"rendered":"<p><a class=\"aligncenter blog-photo\" href=\"https:\/\/lifeboat.com\/blog.images\/a-boost-for-the-precision-of-genome-editing2.jpg\"><\/a><\/p>\n<p>The FDA\u2019s recent approval of the first CRISPR-Cas9\u2013based gene therapy has marked a major milestone in biomedicine, validating genome editing as a promising treatment strategy for disorders like sickle cell disease, muscular dystrophy, and certain cancers.<\/p>\n<p>CRISPR-Cas9, often likened to \u201cmolecular scissors,\u201d allows scientists to cut DNA at targeted sites to snip, repair, or replace genes. But despite its power, Cas9 poses a critical safety risk: the active enzyme can linger in cells and cause unintended DNA breaks\u2014so-called off-target effects\u2014which may trigger harmful mutations in healthy genes.<\/p>\n<p>Now, researchers in the labs of Professor Ronald T. Raines (MIT Department of Chemistry) and Professor Amit Choudhary (Harvard Medical School) have engineered a precise way to turn Cas9 off after its job is done\u2014significantly reducing off-target effects and improving the clinical safety of gene editing. Their findings are detailed in a new paper published this week in the <i>Proceedings of the National Academy of Sciences<\/i> (PNAS).<\/p>\n<hr>\n<p>Researchers used cells that glow green due to a green fluorescent protein (GFP) gene. If Cas9 is working, it disrupts the GFP gene and the cells stop glowing. If LFN-Acr blocks Cas9, the cells keep glowing. These images depict cells in different conditions: some with active Cas9 (which stopped the green glow), some with Cas9 and LFN-Acr (glow stayed on).<\/p>\n<p>Image courtesy of the researchers.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>The FDA\u2019s recent approval of the first CRISPR-Cas9\u2013based gene therapy has marked a major milestone in biomedicine, validating genome editing as a promising treatment strategy for disorders like sickle cell disease, muscular dystrophy, and certain cancers. CRISPR-Cas9, often likened to \u201cmolecular scissors,\u201d allows scientists to cut DNA at targeted sites to snip, repair, or replace [\u2026]<\/p>\n","protected":false},"author":534,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1902,11,412],"tags":[],"class_list":["post-219742","post","type-post","status-publish","format-standard","hentry","category-bioengineering","category-biotech-medical","category-genetics"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/219742","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/users\/534"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=219742"}],"version-history":[{"count":0,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/219742\/revisions"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=219742"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=219742"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=219742"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}