{"id":9317,"date":"2022-06-15T06:46:59","date_gmt":"2022-06-15T06:46:59","guid":{"rendered":"https:\/\/www.booksofall.com\/?post_type=product&#038;p=9317"},"modified":"2022-06-15T06:46:59","modified_gmt":"2022-06-15T06:46:59","slug":"gene-therapies-only-work-for-some-people-so-how-do-we-fix-this","status":"publish","type":"product","link":"https:\/\/www.booksofall.com\/pt\/gene-therapies-only-work-for-some-people-so-how-do-we-fix-this\/","title":{"rendered":"Gene therapies only work for some people \u2013 so how do we fix this?"},"content":{"rendered":"<h4 id=\"standfirst\" class=\"article__standfirst\">Gene therapies could offer long-term fixes for a host of incurable illnesses \u2013 except that many people\u2019s immune systems react to them. So researchers are looking for ways to help the treatments sneak past our antibodies.<\/h4>\n<aside class=\"article__aside\">\n<div class=\"article__aside__section article__share-buttons\" role=\"group\">\n<p>Growing up in Devon in the 1980s, brothers James* and Matt* had to be careful with their antics \u2013 a cut or bruise could land them in hospital.<\/p>\n<p>They have haemophilia, a genetic disease where the blood doesn\u2019t clot efficiently due to a lack of a clotting protein. It\u2019s plagued their family for generations. Their granny\u2019s brother died from it, and they, their uncle and a younger cousin have long managed it by injecting the missing protein to prevent bleeds.<\/p>\n<p>While haemophilia can now be managed effectively \u2013 in high-income countries at least, where medication is readily available \u2013 it can severely limit freedom and quality of life and can even reduce life expectancy. Bleeds can be triggered by minor strains that healthy people barely notice, such as from walking or lifting boxes. Blood then seeps from the capillaries and pools in the joints, causing throbbing pain and lasting damage.<\/p>\n<p>Although the younger of the two at 41, Matt\u2019s joints are the more wrecked; he can\u2019t straighten his right arm due to eroded cartilage in the elbow, while both his ankles and his right knee are in bad shape. For James, 45, it\u2019s worst in his ankles, which swell up and bleed after walking or standing for too long.<\/p>\n<p>So when they heard about a clinical trial for an experimental gene therapy, both brothers were intrigued. Unlike the injections, the therapy might fix their haemophilia for good. It\u2019s a simple idea: if you inherit a faulty gene, then putting a working copy into the body\u2019s cells should fix things.<\/p>\n<h4>Reference:<\/h4>\n<ul>\n<li><a href=\"https:\/\/ghr.nlm.nih.gov\/primer\/therapy\/genetherapy\">This explainer<\/a>, published by\u00a0the US National Library of Medicine, outlines what gene therapy is and how it works, and offers links to\u00a0further information.<\/li>\n<li>The\u00a0<em>New England Journal of Medicine<\/em>\u00a0<a href=\"https:\/\/www.nejm.org\/doi\/full\/10.1056\/NEJMoa1708483#t=article\">write-up<\/a>\u00a0of the trial Jaems took part in. The trial\u2019s success was also covered in the\u00a0<em><a href=\"https:\/\/www.theguardian.com\/science\/2017\/dec\/14\/leap-forward-towards-gene-therapy-cure-for-haemophilia-a\">Guardian<\/a><\/em>.<\/li>\n<li>This\u00a0<a href=\"https:\/\/www.wired.com\/2013\/08\/the-fall-and-rise-of-gene-therapy-2\/\"><em>Wired<\/em>\u00a0article<\/a>\u00a0plots the fall and rise of gene therapy following the death of Jesse Gelsinger.<\/li>\n<li>In this\u00a0<a href=\"https:\/\/www.ncbi.nlm.nih.gov\/pmc\/articles\/PMC4442590\/\">2015 retrospective<\/a>, Jude Samulski and Eric Hastie assess the progress made since the discovery of AAV over half a century ago.<\/li>\n<\/ul>\n<\/div>\n<\/aside>\n","protected":false},"excerpt":{"rendered":"<p><iframe style=\"width: 100%; height: 750px; border: none;\" src=\"https:\/\/online.visual-paradigm.com\/share\/book\/gene-therapies-only-work-for-some-people-so-how-do-we-fix-this-zl66wi05j?enforceShowPromotionBar=true\" frameborder=\"0\" allowfullscreen=\"allowfullscreen\"><\/iframe><\/p>\n","protected":false},"featured_media":9327,"template":"","meta":{"_yoast_wpseo_title":"","_yoast_wpseo_metadesc":"Design flipbooks online. Keep the book content up-to-date with the instant update tool. Try Visual Paradigm Online, the free flipbook maker."},"product_brand":[],"product_cat":[15,222],"product_tag":[],"class_list":{"0":"post-9317","1":"product","2":"type-product","3":"status-publish","4":"has-post-thumbnail","6":"product_cat-all","7":"product_cat-anthropology","9":"first","10":"instock","11":"shipping-taxable","12":"product-type-simple"},"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.1.1 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Gene therapies only work for some people \u2013 so how do we fix this? - BooksOfAll Portuguese<\/title>\n<meta name=\"description\" content=\"Design flipbooks online. Keep the book content up-to-date with the instant update tool. 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