![\"sickle](\"https:\/\/www.europesays.com\/uk\/wp-content\/uploads\/2025\/06\/shutterstock_2221001799-750x500.jpg\")
<\/p>\nResearchers have developed a novel gene therapy approach that reactivates dormant genes by repositioning them closer to genetic switches called enhancers \u2013 showing promise for treating blood disorders like sickle cell disease.<\/p>\n
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In a new advancement for gene therapy, researchers from the Hubrecht Institute (De Laat group)<\/a>, Erasmus MC<\/a>, and Sanquin<\/a> have developed a new method that can reactivate dormant genes by altering their proximity to powerful genetic switches \u2013 called enhancers. Their approach, dubbed \u2018delete-to-recruit\u2019, uses CRISPR-Cas9 to remove the DNA segment separating a gene from its enhancer \u2013 reviving gene activity without introducing any foreign genetic material.<\/p>\n The findings, published in the journal Blood<\/a>, demonstrate the technique\u2019s potential to treat genetic diseases by restoring function to genes that are otherwise inactive in adulthood.<\/p>\n How enhancers control gene activity<\/p>\n Genes carry the instructions for producing proteins \u2013 the essential workers of our cells. However, not all genes are switched on at the same time. Many genes are tightly regulated, turning on only during specific life stages or under certain conditions. Enhancers, which act like genetic switches, help control this regulation by turning genes on and off.<\/p>\n \u201cEnhancers can be located next to the gene they control, but can also be far away on the DNA,\u201d explained Anna-Karina Felder, one of the study\u2019s first authors. \u201cIn this study, we discovered that it\u2019s possible to activate a gene by bringing it closer to an enhancer.\u201d<\/p>\n CRISPR: cutting the distance<\/p>\n To manipulate the distance between a gene and its enhancer, the team used CRISPR-Cas9, a molecular tool that acts like highly precise genetic scissors.<\/p>\n \u201cWe directed the scissors to cut out a piece of DNA between an enhancer and its gene, bringing them closer together,\u201d said Felder. \u201cIn adult cells, this successfully reactivated genes that are normally only active during embryonic development.\u201d The researchers call this approach \u2018delete-to-recruit\u2019, describing it as a completely new way to turn on dormant genes.<\/p>\n Treating sickle cell disease and beta-thalassemia<\/p>\n The team focused their study on two inherited blood disorders \u2013 sickle cell disease<\/a> and beta-thalassemia. Both conditions are caused by faults in the adult globin gene, which is responsible for producing haemoglobin. The faulty gene results in malformed red blood cells that break down prematurely, causing chronic anaemia, fatigue and long-term organ damage.<\/p>\n In these cases, the foetal version of the globin gene could provide a workaround.<\/p>\n \u201cIn people with sickle cell disease or beta-thalassemia, it\u2019s the adult globin gene \u2013 the main engine that powers red blood cells \u2013 that is broken. But foetal globin is like a backup engine,\u201d said Felder. \u201cBy switching it back on, we can repower the red blood cells and possibly cure these patients.\u201d<\/p>\n Validated in human blood stem cells<\/p>\n The researchers collaborated with Erasmus MC (Philipsen group) and Sanquin (Van den Akker group) to test the method in both healthy donor cells and cells from patients with sickle cell disease. They confirmed that the technique worked in blood stem cells, which are responsible for generating all types of blood cells, including red blood cells.<\/p>\n By reactivating foetal globin in these stem cells, the therapy could potentially generate a continuous supply of healthy red blood cells in patients, offering a long-lasting solution to their condition.<\/p>\n A broader impact for gene therapy<\/p>\n While the research is still in its early phases, its implications are vast.<\/p>\n While we\u2019re still in the early stages, this research lays important groundwork for the development of new gene therapies<\/p>\n \u201cWhile we\u2019re still in the early stages, this research lays important groundwork for the development of new gene therapies,\u201d said Felder. She added that the technique could extend beyond blood diseases to any condition where reactivating a \u2018backup\u2019 gene could compensate for a malfunctioning one.<\/p>\n \u201cEditing the distance to an enhancer, instead of the genes themselves, could offer a versatile therapeutic approach,\u201d she concluded.<\/p>\n A potentially safer, more accessible alternative<\/p>\n Although a gene therapy for sickle cell disease was approved in Europe in 2024, its high cost and complex mechanism limit its use. That treatment involves editing a repressor gene to reactivate foetal globin, but it may also affect other genes in unpredictable ways.<\/p>\n The delete-to-recruit method could offer a safer and more cost-effective alternative. By repositioning DNA without adding or altering genes \u2013 it may reduce risks and increase accessibility for patients around the world.<\/p>\n Related topics
Clinical Trials<\/a>, CRISPR<\/a>, Drug Discovery Processes<\/a>, Epigenetics<\/a>, Gene Therapy<\/a>, Genetic Analysis<\/a>, Genome Editing<\/a>, Molecular Biology<\/a>, Molecular Targets<\/a>, Precision Medicine<\/a>, Translational Science<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"Researchers have developed a novel gene therapy approach that reactivates dormant genes by repositioning them closer to genetic…\n","protected":false},"author":2,"featured_media":213482,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[3846],"tags":[267,70,16,15],"class_list":{"0":"post-213481","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-genetics","8":"tag-genetics","9":"tag-science","10":"tag-uk","11":"tag-united-kingdom"},"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@uk\/114744742653974148","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/213481","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/comments?post=213481"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/213481\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media\/213482"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media?parent=213481"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/categories?post=213481"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/tags?post=213481"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}