{"id":1338496,"date":"2024-08-08T00:17:32","date_gmt":"2024-08-07T23:17:32","guid":{"rendered":"https:\/\/www.businesstelegraph.co.uk\/first-gene-editing-therapy-may-cure-blood-disorder\/"},"modified":"2024-08-08T00:17:32","modified_gmt":"2024-08-07T23:17:32","slug":"first-gene-editing-therapy-may-cure-blood-disorder","status":"publish","type":"post","link":"https:\/\/www.businesstelegraph.co.uk\/first-gene-editing-therapy-may-cure-blood-disorder\/","title":{"rendered":"First gene-editing therapy may cure blood disorder"},"content":{"rendered":"<p> <script async src=\"\/\/pagead2.googlesyndication.com\/pagead\/js\/adsbygoogle.js\"><\/script>\r\n<!-- newscabal uk text -->\r\n<ins class=\"adsbygoogle\"\r\n     style=\"display:inline-block;width:728px;height:90px\"\r\n     data-ad-client=\"ca-pub-8162386273903324\"\r\n     data-ad-slot=\"3688094655\"><\/ins>\r\n<script>\r\n(adsbygoogle = window.adsbygoogle || []).push({});\r\n<\/script><br \/>\n<\/p>\n<div id=\"\">\n<figure>\n<div data-component=\"image-block\" class=\"sc-18fde0d6-0 ejjhCR\">\n<div data-testid=\"hero-image\" class=\"sc-814e9212-1 fcEyBx\"><img decoding=\"async\" src=\"https:\/\/www.businesstelegraph.co.uk\/wp-content\/uploads\/2024\/05\/After-Life-actress-says-making-music-has-been-healing.png\" class=\"sc-814e9212-0 cCvKR hide-when-no-script\"\/><img decoding=\"async\"   src=\"http:\/\/www.businesstelegraph.co.uk\/wp-content\/uploads\/2024\/08\/First-gene-editing-therapy-may-cure-blood-disorder.webp.webp\" loading=\"eager\" alt=\"Kirthana Balachandran Kirthana Balachandran\" class=\"sc-814e9212-0 hIXOPW\"\/><span class=\"sc-814e9212-2 jesyMJ\">Kirthana Balachandran<\/span><\/div>\n<\/div>\n<p><figcaption class=\"sc-8353772e-0 cvNhQw\">Kirthana Balachandran was diagnosed when she was three months old and is now studying to be a doctor<!-- --><\/figcaption><\/p>\n<\/figure>\n<div data-component=\"text-block\" class=\"sc-18fde0d6-0 dlWCEZ\">\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">The first therapy that uses gene-editing is to be offered on the NHS in a \u201crevolutionary breakthrough\u201d for patients.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">It will be used as a potential cure for the blood disorder beta thalassaemia.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">Stem cells which make blood will be extracted, reprogrammed to correct the condition and returned to the patient&#8217;s body. <!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">It could spare them needing a blood transfusion, every three to five weeks, for life.<!-- --><\/p>\n<\/div>\n<div data-component=\"text-block\" class=\"sc-18fde0d6-0 dlWCEZ\">\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">People with beta thalassaemia struggle to produce enough haemoglobin, which is the protein in red blood cells that carries oxygen around the body.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">It is a genetic disease that is passed down through families and caused by defects in the body&#8217;s instructions for manufacturing haemoglobin.  <!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">It can leave people severely tired, weak, and short of breath and also cuts life expectancy.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">Kirthana Balachandran, 21, was diagnosed when she was only three months old and gets muscle pain and back pain and can have palpitations when walking uphill.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">\u201cThe idea of depending on transfusions for quite literally the rest of my life is daunting, I constantly worry about the future,\u201d she says.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">The idea of gene-editing is you only need to do it once.<!-- --><\/p>\n<\/div>\n<p><h2 class=\"sc-518485e5-0 kRvAla\">How the gene-editing works<!-- --><\/h2>\n<\/p>\n<div data-component=\"text-block\" class=\"sc-18fde0d6-0 dlWCEZ\">\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">It relies on a tool called Crispr, which won the Nobel Prize for Chemistry in 2020. It is essentially a satnav connected to a pair of scissors \u2013 one part targets the right section of DNA and the other performs the edit.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">You might think this technology is being used to repair the genetic defect, but it is actually cuter than that. <!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">Instead it relies on the fact your body makes different types of haemoglobin before and after birth.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">When we are still in the womb, our bodies use &#8220;fetal haemoglobin&#8221; to pull oxygen out of our mother&#8217;s bloodstream.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">Once we are born, a genetic switch is flipped and we start making &#8220;adult haemoglobin&#8221;. <!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">Crucially, it is only the adult form of haemoglobin that is affected by beta thalassaemia.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">So the therapy disables the switch &#8211; named BCL11A \u2013 so the adult body starts making fetal haemoglobin once again. <!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">In order to do this, the stem cells that manufacture red blood cells in our spongy bone marrow are harvested.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">These cells are sent to the lab where the genetic switch is targeted.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">The next stage is unpleasant. It takes a course of chemotherapy to kill off the old stem cells that were producing broken haemoglobin, before the new ones can be put in.<!-- --><\/p>\n<\/div>\n<figure>\n<div data-component=\"image-block\" class=\"sc-18fde0d6-0 gfTVSf\">\n<div data-testid=\"image\" class=\"sc-814e9212-1 fcEyBx\"><img decoding=\"async\" src=\"https:\/\/www.businesstelegraph.co.uk\/wp-content\/uploads\/2024\/05\/After-Life-actress-says-making-music-has-been-healing.png\" class=\"sc-814e9212-0 cCvKR hide-when-no-script\"\/><img decoding=\"async\"   src=\"http:\/\/www.businesstelegraph.co.uk\/wp-content\/uploads\/2024\/08\/1723123337_109_First-gene-editing-therapy-may-cure-blood-disorder.webp.webp\" loading=\"lazy\" alt=\"Graphic explaining the process of extracting bone marrow, editing it in the lab and putting it back into the patient\" class=\"sc-814e9212-0 hIXOPW\"\/><\/div>\n<\/div>\n<\/figure>\n<div data-component=\"text-block\" class=\"sc-18fde0d6-0 dlWCEZ\">\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">Abdul-Qadeer Akhtar, 28 and from Hemel Hempstead, took part in clinical trials in 2020.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">He said the treatment was \u201cchallenging\u201d and \u201ctough\u201d but since having it, he has been \u201chealthier and more active\u201d.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">\u201c[I] have even taken up boxing, I can travel more freely now, which is fantastic, I\u2019m eager to embrace life to the fullest.\u201d<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\"><a target=\"_blank\" href=\"https:\/\/news.vrtx.com\/news-releases\/news-release-details\/vertex-presents-positive-long-term-data-casgevytm-exagamglogene\" class=\"sc-c9299ecf-0 bZUiKB\" rel=\"noopener\">Data shows<!-- --><\/a> that out of 52 patients given the treatment, 49 did not need another blood transfusion for at least a year of monitoring. The approach is so new that the long-term effects are still unknown. <!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">Kirthana says: \u201cWith gene therapy, I would potentially have a cure, and not have to have my three-weekly transfusions.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">\u201cIt would be a life-changing treatment.\u201d<!-- --><\/p>\n<\/div>\n<p><h2 class=\"sc-518485e5-0 kRvAla\">A \u00a31.6m price tag?<!-- --><\/h2>\n<\/p>\n<div data-component=\"text-block\" class=\"sc-18fde0d6-0 dlWCEZ\">\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">The National Institute of Health and Care Excellence medicines watchdog has approved the therapy after assessing the costs and benefits of what it calls a \u201cpotential cure\u201d.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">A deal has been struck which means NHS England is paying less than the official price of \u00a31.6m per patient.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">It is estimated that 460 people over the age of 12 would be eligible if they wanted the therapy, and it will be offered at seven specialist centres \u201cwithin weeks\u201d.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">Amanda Pritchard, the NHS chief executive, said: \u201cThis is a historic moment for people living with beta thalassaemia with a potential cure for those facing this debilitating disorder now available on the NHS.\u201d<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">Beta thalassemia mainly affects people of Mediterranean, south Asian, south-east Asian and Middle Eastern backgrounds.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">Previously the only alternative to blood transfusions was a stem cell transplant, but this was rare because it required a very close tissue match from a donor. <!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">Casgevy, developed by the company Vertex, is the first approved therapy to use Crispr-technology.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">Romaine Maharaj, executive director of the UK Thalassaemia Society, said \u201cwe stand on the brink of a revolutionary breakthrough\u201d and \u201cit is a beacon of hope\u201d.<!-- --><\/p>\n<p class=\"sc-eb7bd5f6-0 fYAfXe\">Negotiations are still under way to see if the same therapy can be used on the NHS for another genetic disease affecting haemoglobin &#8211; sickle cell anaemia. <!-- --><\/p>\n<\/div>\n<\/div>\n<p><script async src=\"\/\/pagead2.googlesyndication.com\/pagead\/js\/adsbygoogle.js\"><\/script>\r\n<ins class=\"adsbygoogle\"\r\n     style=\"display:block; text-align:center;\"\r\n     data-ad-layout=\"in-article\"\r\n     data-ad-format=\"fluid\"\r\n     data-ad-client=\"ca-pub-8162386273903324\"\r\n     data-ad-slot=\"7859258044\"><\/ins>\r\n<script>\r\n     (adsbygoogle = window.adsbygoogle || []).push({});\r\n<\/script><br \/>\n<br \/><a href=\"https:\/\/www.bbc.com\/news\/articles\/c4gzldll44lo\">READ SOURCE<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Kirthana Balachandran Kirthana Balachandran was diagnosed when she was three months old and is now studying to be a doctor The first therapy that uses gene-editing is to be offered on the NHS in a \u201crevolutionary breakthrough\u201d for patients. It will be used as a potential cure for the blood disorder beta thalassaemia. Stem cells [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":1338497,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_oct_exclude_from_cache":false,"footnotes":""},"categories":[11],"tags":[],"class_list":["post-1338496","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-health"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v24.1 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>First gene-editing therapy may cure blood disorder - Business Telegraph<\/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.bbc.com\/news\/articles\/c4gzldll44lo\" \/>\n<meta property=\"og:locale\" content=\"en_GB\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"First gene-editing therapy may cure blood disorder - Business Telegraph\" \/>\n<meta property=\"og:description\" content=\"Kirthana Balachandran Kirthana Balachandran was diagnosed when she was three months old and is now studying to be a doctor The first therapy that uses gene-editing is to be offered on the NHS in a \u201crevolutionary breakthrough\u201d for patients. It will be used as a potential cure for the blood disorder beta thalassaemia. Stem cells [&hellip;]\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.bbc.com\/news\/articles\/c4gzldll44lo\" \/>\n<meta property=\"og:site_name\" content=\"Business Telegraph\" \/>\n<meta property=\"article:published_time\" content=\"2024-08-07T23:17:32+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/www.businesstelegraph.co.uk\/wp-content\/uploads\/2024\/08\/First-gene-editing-therapy-may-cure-blood-disorder.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"1024\" \/>\n\t<meta property=\"og:image:height\" content=\"576\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"author\" content=\"businesstelegraph\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"businesstelegraph\" \/>\n\t<meta name=\"twitter:label2\" content=\"Estimated reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"4 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\/\/www.bbc.com\/news\/articles\/c4gzldll44lo#article\",\"isPartOf\":{\"@id\":\"https:\/\/www.businesstelegraph.co.uk\/first-gene-editing-therapy-may-cure-blood-disorder\/\"},\"author\":{\"name\":\"businesstelegraph\",\"@id\":\"https:\/\/www.businesstelegraph.co.uk\/#\/schema\/person\/0a3a6a0f58402f1f2af94e02d0987b78\"},\"headline\":\"First gene-editing therapy may cure blood disorder\",\"datePublished\":\"2024-08-07T23:17:32+00:00\",\"dateModified\":\"2024-08-07T23:17:32+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\/\/www.businesstelegraph.co.uk\/first-gene-editing-therapy-may-cure-blood-disorder\/\"},\"wordCount\":770,\"publisher\":{\"@id\":\"https:\/\/www.businesstelegraph.co.uk\/#organization\"},\"image\":{\"@id\":\"https:\/\/www.bbc.com\/news\/articles\/c4gzldll44lo#primaryimage\"},\"thumbnailUrl\":\"https:\/\/www.businesstelegraph.co.uk\/wp-content\/uploads\/2024\/08\/First-gene-editing-therapy-may-cure-blood-disorder.jpg\",\"articleSection\":[\"Health\"],\"inLanguage\":\"en-GB\"},{\"@type\":\"WebPage\",\"@id\":\"https:\/\/www.businesstelegraph.co.uk\/first-gene-editing-therapy-may-cure-blood-disorder\/\",\"url\":\"https:\/\/www.bbc.com\/news\/articles\/c4gzldll44lo\",\"name\":\"First gene-editing therapy may cure blood disorder - 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It will be used as a potential cure for the blood disorder beta thalassaemia. 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