{"id":34723,"date":"2018-10-31T13:03:07","date_gmt":"2018-10-31T07:33:07","guid":{"rendered":"https:\/\/blog.forumias.com\/?p=34723"},"modified":"2018-10-31T13:03:07","modified_gmt":"2018-10-31T07:33:07","slug":"in-a-first-gene-therapy-halts-a-fatal-brain-disease","status":"publish","type":"post","link":"https:\/\/forumias.com\/blog\/in-a-first-gene-therapy-halts-a-fatal-brain-disease\/","title":{"rendered":"In a first, gene therapy halts a fatal brain disease:"},"content":{"rendered":"<p><a href=\"https:\/\/www.google.co.in\/url?sa=t&amp;rct=j&amp;q=&amp;esrc=s&amp;source=web&amp;cd=1&amp;cad=rja&amp;uact=8&amp;ved=0ahUKEwjKvNbXpeDWAhVIP48KHXlWB1cQFgglMAA&amp;url=http%3A%2F%2Fwww.thehindu.com%2Ftodays-paper%2Ftp-opinion%2Fin-a-first-gene-therapy-halts-a-fatal-brain-disease%2Farticle19821943.ece&amp;usg=AOvVaw2q5vN8y-LzNovX7qIWct0S\"><span style=\"font-weight: 400;\">In a first, gene therapy halts a fatal brain disease<\/span><\/a><span style=\"font-weight: 400;\">:<\/span><\/p>\n<p><b>Context:<\/b><\/p>\n<ul>\n<li style=\"font-weight: 400;\"><span style=\"font-weight: 400;\">A new study indicates that gene therapy can hold off Adrenoleukodystrophy (ALD), a rare disorder, without side effects, but only if it is begun when the only signs of deterioration are changes in brain scans.<\/span><\/li>\n<li style=\"font-weight: 400;\"><span style=\"font-weight: 400;\">The key to making the therapy work is human Immunodeficiency virus (HIV).<\/span><\/li>\n<\/ul>\n<p><b>Was the study successful?<\/b><\/p>\n<ul>\n<li style=\"font-weight: 400;\"><span style=\"font-weight: 400;\">The study involved 17 boys aged 4 to 13. All got gene therapy. Two years later, 15 were functioning normally without obvious symptoms.<\/span><\/li>\n<li style=\"font-weight: 400;\"><span style=\"font-weight: 400;\">The study opened up new avenues for using gene therapy to treat brain diseases.<\/span><\/li>\n<\/ul>\n<p><b>What is ALD?<\/b><\/p>\n<ul>\n<li style=\"font-weight: 400;\"><span style=\"font-weight: 400;\">A mutated gene causes a rare disorder, adrenoleukodystrophy, or ALD.<\/span><\/li>\n<li style=\"font-weight: 400;\"><span style=\"font-weight: 400;\">Nerve cells in the brain die, and in a few short years, children lose the ability to walk or talk.<\/span><\/li>\n<li style=\"font-weight: 400;\"><span style=\"font-weight: 400;\">They become unable to eat without a feeding tube, to see, hear or think. They usually die within five years of diagnosis.<\/span><\/li>\n<li style=\"font-weight: 400;\"><span style=\"font-weight: 400;\">The disease strikes about one in 20,000 boys; symptoms first occur at an average age of 7<\/span><\/li>\n<\/ul>\n<p><b>Is there any cure of the disorder?<\/b><\/p>\n<ul>\n<li style=\"font-weight: 400;\"><span style=\"font-weight: 400;\">The only treatment is a bone-marrow transplant, if a compatible donor can be found or a transplant with cord blood, if it was saved at birth.<\/span><\/li>\n<li style=\"font-weight: 400;\"><span style=\"font-weight: 400;\">These transplants are difficult and dangerous therapy, with a mortality rate as high as 20%. Some who survive are left with lifelong disabilities.<\/span><\/li>\n<\/ul>\n","protected":false},"excerpt":{"rendered":"<p>In a first, gene therapy halts a fatal brain disease: Context: A new study indicates that gene therapy can hold off Adrenoleukodystrophy (ALD), a rare disorder, without side effects, but only if it is begun when the only signs of deterioration are changes in brain scans. The key to making the therapy work is human&hellip; <a class=\"more-link\" href=\"https:\/\/forumias.com\/blog\/in-a-first-gene-therapy-halts-a-fatal-brain-disease\/\">Continue reading <span class=\"screen-reader-text\">In a first, gene therapy halts a fatal brain disease:<\/span><\/a><\/p>\n","protected":false},"author":61,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"jetpack_post_was_ever_published":false,"footnotes":""},"categories":[555],"tags":[],"class_list":["post-34723","post","type-post","status-publish","format-standard","hentry","category-test-1","entry"],"jetpack_featured_media_url":"","views":{"total":0,"cached_at":"","cached_date":1704957742},"jetpack_sharing_enabled":true,"_links":{"self":[{"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/posts\/34723","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/users\/61"}],"replies":[{"embeddable":true,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/comments?post=34723"}],"version-history":[{"count":0,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/posts\/34723\/revisions"}],"wp:attachment":[{"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/media?parent=34723"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/categories?post=34723"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/tags?post=34723"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}