![\"HRI\"](\"https:\/\/www.europesays.com\/uk\/wp-content\/uploads\/2025\/08\/sickle-cell-750x450.gif\")
<\/p>\nResearchers at UNSW Sydney have unveiled a next-generation CRISPR tool that could lead to safer treatments for genetic disorders, including Sickle Cell.<\/p>\n
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A new generation of CRISPR technology developed at UNSW Sydney<\/a> offers a safer path to treating genetic diseases like Sickle Cell<\/a>, while also proving beyond doubt that chemical tags on DNA \u2013 often thought to be little more than genetic cobwebs \u2013 actively silence genes.<\/p>\n For years, scientists have debated whether methyl groups \u2013 small chemical clusters that accumulate on DNA \u2013 are simply waste that collects in the genome where genes are turned off.<\/p>\n But now researchers at UNSW and the St Jude Children\u2019s Research Hospital (Memphis)<\/a>, have published a study in Nature Communications<\/a> that reveals that removing these tags can switch genes back on \u2013 confirming that methylation is not just correlated with silencing, but directly responsible for it.<\/p>\n \u201cWe showed very clearly that if you brush the cobwebs off, the gene comes on,\u201d says study lead author Professor Merlin Crossley, UNSW Deputy Vice-Chancellor Academic Quality.<\/p>\n \u201cAnd when we added the methyl groups back to the genes, they turned off again. So, these compounds aren\u2019t cobwebs \u2013 they\u2019re anchors.\u201d<\/p>\n What is CRISPR?<\/p>\n CRISPR \u2013 otherwise known as clustered regularly interspaced short palindromic repeats \u2013 forms the basis of gene-editing technology that allows scientists to find and change faulty sections of DNA \u2013 often by replacing them with healthy ones.<\/p>\n It uses a naturally occurring process, first observed in bacteria fighting off invading viruses by cutting the virus DNA strands.<\/p>\n The first generation of CRISPR tools worked in this way, by cutting DNA sequences to disable faulty genes. The second generation allowed researchers to zoom in and correct individual letters in the genetic code. But both approaches involved making cuts to the genetic code, which comes with the risk of unwanted changes that could cause other health problems.<\/p>\n The third generation \u2013 known as epigenetic editing \u2013 instead looks at the surface of genes. Rather than cutting DNA strands to remove or edit faulty genes \u2013 this method removes methyl groups attached to silenced or suppressed genes.<\/p>\n A potential breakthrough for sickle cell disease<\/p>\n The researchers say epigenetic editing could be used to treat people affected by Sickle Cell-related diseases, which are genetic mutations that alter the shape and function of red blood cells \u2013 leading to chronic pain, organ damage and reduced life expectancy.<\/p>\n \u201cWhenever you cut DNA, there\u2019s a risk of cancer, and if you\u2019re doing a gene therapy for a lifelong disease, that\u2019s a bad kind of risk,\u201d Professor Crossley says.<\/p>\n \u201cBut if we can do gene therapy that doesn\u2019t involve snipping DNA strands, then we avoid these potential pitfalls.\u201d<\/p>\n Instead of cutting, the new method uses a modified CRISPR system to deliver enzymes that remove methyl groups from DNA \u2013 effectively lifting the brakes on silenced genes. The foetal globin gene plays a key role in delivering oxygenated blood to a developing foetus in utero, and the researchers say switching it back on following birth could provide a workaround for the faulty gene that causes Sickle Cell diseases.<\/p>\n \u201cYou can think of the foetal globin gene as the training wheels on a kid\u2019s bike,\u201d says Crossley. \u201cWe believe we can get them working again in people who need new wheels.\u201d<\/p>\n The big picture<\/p>\n So far, all work to achieve this has been carried out in a lab on human cells at UNSW and in Memphis.<\/p>\n Study co-author Professor Kate Quinlan says the discovery is not only promising for people with Sickle Cell disease, but also for other genetic diseases where turning certain genes on or off by altering methyl groups avoids having to cut DNA strands.<\/p>\n \u201cWe are excited about the future of epigenetic editing as our study shows that it allows us to boost gene expression without modifying the DNA sequence. Therapies based on this technology are likely to have a reduced risk of unintended negative effects compared to first or second generation CRISPR,\u201d she says.<\/p>\n In the future, once testing in animals and clinical trials are complete, doctors could use the method to treat Sickle Cell disease by first collecting some of the patient\u2019s blood stem cells. In a lab, epigenetic editing would be used to remove the methyl chemical tags from the foetal globin gene to reactivate it. The edited cells would then be returned to the patient, where they settle back into the bone marrow and start producing healthier blood cells.<\/p>\n","protected":false},"excerpt":{"rendered":"Researchers at UNSW Sydney have unveiled a next-generation CRISPR tool that could lead to safer treatments for genetic…\n","protected":false},"author":2,"featured_media":354368,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[3846],"tags":[267,70,16,15],"class_list":{"0":"post-354367","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\/115050279500587260","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/354367","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=354367"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/354367\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media\/354368"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media?parent=354367"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/categories?post=354367"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/tags?post=354367"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}