{"id":30196,"date":"2024-10-01T21:48:00","date_gmt":"2024-10-01T21:48:00","guid":{"rendered":"https:\/\/www.prnasia.com\/story\/archive\/4520052_AE20052_0"},"modified":"2024-10-01T21:48:00","modified_gmt":"2024-10-01T21:48:00","slug":"australia-takes-centre-stage-as-a-cure-for-rett-syndrome-draws-closer","status":"publish","type":"post","link":"https:\/\/transmediavictoria.net.au\/?p=30196","title":{"rendered":"Australia takes centre stage as a cure for Rett Syndrome draws closer"},"content":{"rendered":"<p><span class=\"legendSpanClass\"><span class=\"xn-location\">GOLD COAST, Australia<\/span><\/span>, <span class=\"legendSpanClass\"><span class=\"xn-chron\">Oct. 2, 2024<\/span><\/span> \/PRNewswire\/ &#8212; Australian researcher Associate Professor <span class=\"xn-person\">Wendy Gold<\/span>, a world leader in <a href=\"https:\/\/youtu.be\/j0NjejPBrPM\" target=\"_blank\" rel=\"nofollow noopener\">Rett Syndrome<\/a> research from the <span class=\"xn-org\">University of Sydney<\/span>, joins international colleagues and Rett families on the <span class=\"xn-location\">Gold Coast<\/span> today to present the latest advances toward a cure.<\/p>\n<div class=\"PRN_ImbeddedAssetReference\" id=\"DivAssetPlaceHolder1\" align=\"center\" readability=\"8\">\n<p title=\"What is Rett Syndrome? Associate Professor Wendy Gold explains. Wendy will keynote the World Rett Syndrome Congress this week on the Gold Coast.\"><iframe loading=\"lazy\" width=\"560\" height=\"315\" src=\"https:\/\/www.youtube.com\/embed\/j0NjejPBrPM\" frameborder=\"0\">&amp;amp;amp;amp;amp;amp;nbsp;<\/iframe><br \/><span>What is Rett Syndrome? Associate Professor Wendy Gold explains. Wendy will keynote the World Rett Syndrome Congress this week on the Gold Coast.<\/span><\/p>\n<\/p><\/div>\n<p>Rett Syndrome, which overwhelmingly impacts girls, is a severe genetic disorder. Affecting 1 in 10,000 female babies, sufferers begin to lose their ability to walk and talk at around 18 months of age and require lifelong 24\/7 care. While girls can live into their sixties, they suffer global developmental delay and often also seizures and scoliosis.<\/p>\n<p><a href=\"https:\/\/rettresearch.org.au\/\" target=\"_blank\" rel=\"nofollow noopener\">Rett Research Australia<\/a> founder, <span class=\"xn-person\">Brett Anslow<\/span>, whose 11-year-old daughter Holly has Rett Syndrome, said the Australian Rett community was optimistic heading into the conference, as a cure is closer than ever before.<\/p>\n<p>&#8220;Rett Syndrome is a genetic disorder, so it requires gene therapy to cure it,&#8221; he said. &#8220;Wendy has done some truly extraordinary work toward a cure, as have other researchers from around the world. We feel incredibly hopeful that in the very near future there may be a cure for this absolutely devastating disorder.&#8221;<\/p>\n<p>Associate Professor Gold said her team were well advanced on two fronts looking at ways to cure Rett Syndrome.<\/p>\n<p>&#8220;The first one is a gene editing therapy, which is looking at using what we call &#8216;molecular scissors&#8217; to be placed in the body, to cut out the mutation in the genome and replace it with a normal copy, and we&#8217;ve had some exciting initial success with this,&#8221; she said. &#8220;The other gene therapy we have I can&#8217;t say too much about because we&#8217;re actually looking at protecting it and patenting it, but it is a gene therapy similar to those that are been investigated in clinical trials, but with more regulatory control.&#8221;<\/p>\n<p>Associate Professor Gold and her team, along with working toward a cure, are also developing a diagnostic tool. Today there is no definitive blood test for Rett Syndrome, meaning girls are only treated once symptoms appear. Armed with a diagnostic tool, doctors would be able to treat girls with Rett Syndrome as babies, with the test potentially being able to be added to the standard newborn heel-prick test.<\/p>\n<p>Associate Professor Gold points out that finding a cure for Rett Syndrome would open the floodgates for cures for other genetic diseases.<\/p>\n<p>&#8220;Rett Syndrome is considered the poster child in the rare disease space, and everyone&#8217;s looking at Rett syndrome, watching as they start the clinical trials. Once we have a cure it&#8217;ll just open up so many options for the other disorders. There are 7, 000 rare genetic disorders, so it&#8217;s encouraging to see what happens in the space for Rett syndrome.<\/p>\n<p>Associate Professor Gold said her team had been able to be successful due to the tireless fundraising efforts of the Rett community. &#8220;<span class=\"xn-person\">Brett and Raelene Anslow<\/span> have been absolutely central to the success we have had, by raising money for us to get to a point of success where we could then apply for grants to expand and speed up our work,&#8221; she said. &#8220;90% of grant applications in <span class=\"xn-location\">Australia<\/span> are unsuccessful, and it is only because we have the tireless backing of the Rett community continually fundraising and co-investing in this work, that we can continue moving forward at the pace we need to.&#8221;<\/p>\n<p>Rett experts from <span class=\"xn-location\">Italy<\/span>, the UK, <span class=\"xn-location\">Sweden<\/span>, the US, <span class=\"xn-location\">the Netherlands<\/span>, <span class=\"xn-location\">Canada<\/span>, <span class=\"xn-location\">Israel<\/span>, <span class=\"xn-location\">France<\/span>, <span class=\"xn-location\">Germany<\/span>, <span class=\"xn-location\">Ireland<\/span> and <span class=\"xn-location\">Scotland<\/span> will join Associate Professor Gold and many Rett Syndrome families on the <span class=\"xn-location\">Gold Coast<\/span> from <span class=\"xn-chron\">October 2<\/span><sup>nd<\/sup> \u2013 5<sup>th<\/sup>. For more information about Rett Syndrome or to donate, visit <a href=\"http:\/\/www.rettresearch.org.au\/\" target=\"_blank\" rel=\"nofollow noopener\">www.rettresearch.org.au<\/a>.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>GOLD COAST, Australia, Oct. 2, 2024 \/PRNewswire\/ &#8212; Australian researcher Associate Professor Wendy Gold, a world leader in Rett Syndrome research from the University of Sydney, joins international colleagues and Rett families on the Gold Coast today to present the latest advances toward a cure. &amp;amp;amp;amp;amp;amp;nbsp;What is Rett Syndrome? Associate Professor Wendy Gold explains. Wendy [&hellip;]<\/p>\n","protected":false},"author":8,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[20],"tags":[],"class_list":["post-30196","post","type-post","status-publish","format-standard","hentry","category-business-news"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.9 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Australia takes centre stage as a cure for Rett Syndrome draws closer - My Blog<\/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:\/\/transmediavictoria.net.au\/?p=30196\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Australia takes centre stage as a cure for Rett Syndrome draws closer - My Blog\" \/>\n<meta property=\"og:description\" content=\"GOLD COAST, Australia, Oct. 2, 2024 \/PRNewswire\/ &#8212; Australian researcher Associate Professor Wendy Gold, a world leader in Rett Syndrome research from the University of Sydney, joins international colleagues and Rett families on the Gold Coast today to present the latest advances toward a cure. &amp;amp;amp;amp;amp;amp;nbsp;What is Rett Syndrome? 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