{"id":70575,"date":"2026-02-10T21:03:38","date_gmt":"2026-02-10T10:03:38","guid":{"rendered":"https:\/\/hudson.org.au\/?post_type=hdsn_news_post&#038;p=70575"},"modified":"2026-02-13T14:51:07","modified_gmt":"2026-02-13T03:51:07","slug":"tiny-rna-fragments-unlock-massive-autoimmune-treatment-potential","status":"publish","type":"hdsn_news_post","link":"https:\/\/hudson.org.au\/news\/tiny-rna-fragments-unlock-massive-autoimmune-treatment-potential\/","title":{"rendered":"Tiny RNA fragments unlock massive autoimmune treatment potential"},"content":{"rendered":"\n<p class=\"has-medium-font-size\">RNA-based medicine is about to take a huge leap forward thanks to the discovery of extremely short RNA fragments with important anti-inflammatory properties.<\/p>\n\n\n\n    <div class=\"block--callout flex z-20 text-blue-800 bg-gray-100 p-6 mb-5 sm:mb-8 sm:float-right sm:w-1\/2 sm:ml-6\">\n        <div class=\"cms-content\">\n            <h2 id=\"key-points\">Key Points<\/h2>\n<ul>\n<li>An international study has revealed that tiny fragments of RNA play a critical role in keeping the immune system in check, preventing inflammation<\/li>\n<li>This discovery, published in <em>Nature Immunology<\/em> opens new possibilities for treating autoimmune diseases like lupus, psoriasis and rheumatoid arthritis<\/li>\n<li>Historically, these tiny RNA fragments (between 1 and 3 bases long) were thought to be biological waste<\/li>\n<li>Previous discoveries of short RNA fragments (around 16-20 bases long) have been seen as major scientific advances.<\/li>\n<\/ul>\n        <\/div>\n    <\/div>\n\n    \n\n\n<p><a href=\"https:\/\/hudson.org.au\/researcher-profile\/michael-gantier\/\">Professor Michael Gantier\u2019s<\/a> latest research, published in <em><a href=\"https:\/\/www.nature.com\/articles\/s41590-026-02429-2\" type=\"link\" id=\"https:\/\/www.nature.com\/articles\/s41590-026-02429-2\" target=\"_blank\" rel=\"noreferrer noopener\">Nature Immunology<\/a><\/em>, builds on the Nobel prize winning discoveries of microRNA (2024) and siRNAs (2006), except where those molecules contain around 20 bases, these very short RNA fragments can be as small as 1-3 bases.<\/p>\n\n\n\n<p>So why are these tiny fragments of RNA so important? Prof Gantier explains.<\/p>\n\n\n\n<p>\u201cAfter systematic screening of hundreds of synthetic RNA molecules, we discovered that very short RNA fragments could bind to sensors of our immune system and block their activation,\u201d he said. <\/p>\n\n\n\n<p>\u201cThis is the shortest class of RNAs ever reported to have a biological function (around 6 times shorter than Nobel Prize winning microRNAs!)\u201d.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"h-rna-fragments-potential-for-lupus-and-psoriasis\">RNA fragments potential for lupus and psoriasis<\/h2>\n\n\n\n<p>These findings have enormous significance for the development of new treatments for common autoimmune conditions, such as lupus, psoriasis, rheumatoid arthritis and more.<\/p>\n\n\n\n<p>Prof Gantier\u2019s lab at Hudson Institute of Medical Research in Melbourne led an international team of experts from across Australia, as well as Japan, the UK and the USA.<\/p>\n\n\n\n<p>But they didn\u2019t just identify RNA fragments binding to immune sensors; they also revealed for the first time that they play crucial roles in controlling dangerous <a href=\"https:\/\/hudson.org.au\/disease\/inflammation\/\">inflammation<\/a>, especially in autoimmune conditions.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"h-critical-role-protecting-the-body\">Critical role protecting the body<\/h2>\n\n\n<div class=\"wp-block-image\">\n<figure class=\"alignright size-large\"><a href=\"https:\/\/hudson.org.au\/wp-content\/uploads\/2026\/02\/michael-and-sunil-photo-1-sq.jpg\"><img loading=\"lazy\" decoding=\"async\" width=\"600\" height=\"600\" src=\"https:\/\/hudson.org.au\/wp-content\/uploads\/2026\/02\/michael-and-sunil-photo-1-sq.jpg\" alt=\"L-R: Professor Michael Gantier, Dr Sunil Sapkota\" class=\"wp-image-70576\"\/><\/a><figcaption class=\"wp-element-caption\"><em>L-R: Professor Michael Gantier, Dr Sunil Sapkota<\/em><\/figcaption><\/figure>\n<\/div>\n\n\n<p>With collaborators in Japan the Hudson Institute team solved the puzzle of how these RNA fragments bind to immune receptors at the molecular level.<\/p>\n\n\n\n<p>This revealed something unexpected &#8211; the binding occurred in a new pocket in one of the receptors and blocked its activity. Critically, the team had previously discovered a rare mutation in the same pocket, which led to systemic autoimmunity known as lupus (published in <em>Nature<\/em> in 2022).<\/p>\n\n\n\n<p>The team showed that the rare lupus mutation prevented binding of the short RNA fragments to the pocket of this receptor, meaning the receptor could not be blocked anymore. This suggests that autoimmunity in patients with this pocket mutation originates from an uncontrolled activation of the receptor, thereby revealing the importance of the short RNAs fragments to prevent disease. &nbsp;<\/p>\n\n\n\n<p>\u201cOur discovery shows that selected RNA fragments of only 1-3 bases help protect our bodies against misfiring of the immune system which mistakenly attacks the body &#8211; leading to autoimmunity,\u201d he said.<\/p>\n\n\n\n<p>\u201cThat tells us that extremely short RNA fragments generated during RNA recycling have important anti-inflammatory functions no-one suspected.\u201d<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"h-changing-understanding-of-inflammation\">Changing understanding of inflammation<\/h2>\n\n\n\n<p>\u201cOur findings change the way we understand the initiation and maintenance of chronic inflammation driven by these receptors,\u201d said Prof Gantier. \u201cThey illustrate a mechanism which was entirely unknown &#8211; these receptors are constantly blocked by short RNA fragments and this is essential to maintain health.\u201d<\/p>\n\n\n\n<p>The impact of these findings will only grow as they are further explored.<\/p>\n\n\n\n<p>This work also has direct therapeutic applications for the management of autoimmune diseases, because these tiny RNA fragments can be manufactured to mimic the natural ones and thus prevent aberrant activation of these receptors.<\/p>\n\n\n\n<p>\u201cCurrently, we are particularly targeting autoimmune diseases such as lupus, and its skin manifestation (cutaneous lupus), but we have reason to believe that our approach using short synthetic RNAs could work for many other skin diseases &#8211; including psoriasis,\u201d Prof Gantier said.<\/p>\n\n\n\n<p>\u201cWe are focusing on the skin because of the ease of use of our 3-base RNA fragments in this tissue, though we are also actively working with other companies to develop technologies to target other tissues where autoimmunity is prevalent.\u201d<\/p>\n\n\n\n<figure class=\"wp-block-embed is-type-video is-provider-youtube wp-block-embed-youtube wp-embed-aspect-16-9 wp-has-aspect-ratio\"><div class=\"wp-block-embed__wrapper\">\n<iframe loading=\"lazy\" title=\"RNA Autoimmune Breakthrough\" width=\"640\" height=\"360\" src=\"https:\/\/www.youtube.com\/embed\/od0IT87RbzI?feature=oembed\" frameborder=\"0\" allow=\"accelerometer; autoplay; clipboard-write; encrypted-media; gyroscope; picture-in-picture; web-share\" referrerpolicy=\"strict-origin-when-cross-origin\" allowfullscreen><\/iframe>\n<\/div><figcaption class=\"wp-element-caption\"><em><strong>View video | <\/strong>Professor Michael Gantier and Dr Sunil Sapkota detailing their research<\/em><\/figcaption><\/figure>\n\n\n\n    <div class=\"block--callout flex z-20 text-blue-800 bg-gray-100 p-6 mb-5 sm:mb-8 w-full\">\n        <div class=\"cms-content\">\n            <h2 id=\"related-research-already-making-a-difference\">Related research already making a difference<\/h2>\n<p>Professor Gantier\u2019s findings have recently progressed through their first clinical trial, thanks to Australian-based pharmaceutical company Noxopharm.<\/p>\n<p>The Heracles trial successfully showed the safety of a topical cream containing these tiny RNAs, with the aim of treating skin lupus and other skin autoimmune conditions.<\/p>\n<p>This is the first clinical use of Noxopharm\u2019s SOFRA platform, and the company is confident of its potential for treating a range of autoimmune conditions.<\/p>\n        <\/div>\n    <\/div>\n\n    \n\n\n    <div class=\"block--callout flex z-20 text-blue-800 bg-gray-100 p-6 mb-5 sm:mb-8 w-full\">\n        <div class=\"cms-content\">\n            <h2 id=\"ringing-endorsement\">Ringing endorsement<\/h2>\n<p><em>\u201cThis tour de force from Prof Gantier and his team has transformed our understanding of how immune sensors for viral infections are normally blocked to prevent autoimmunity. Their elegant and fundamental discovery provides a pathway to support the development of new medicines.\u201d<\/em><\/p>\n<p>&#8211; Professor Arthur Krieg, UMass Chan Medical School RNA Therapeutics Institute (USA), one of the world\u2019s foremost experts on oligonucleotides and the immune system, with more than 250 scientific papers and over 50 patents.<\/p>\n        <\/div>\n    <\/div>\n\n    \n\n\n    <div class=\"block--callout flex z-20 text-blue-800 bg-gray-100 p-6 mb-5 sm:mb-8 w-full\">\n        <div class=\"cms-content\">\n            <h2 id=\"about-lupus\">About lupus<\/h2>\n<ul>\n<li>Lupus is a chronic autoimmune disease in which the body\u2019s immune system attacks its own tissues and organs.<\/li>\n<li>More common in women than men, it can inflame the skin, blood cells, joints, kidneys, brain, heart, and lungs.<\/li>\n<li>Lupus is difficult to diagnose as its many symptoms may mimic those of other conditions; they may be mild or cause significant disability.<\/li>\n<li>Some people are born with a tendency to develop lupus, but it can be triggered without warning by an infection, some prescription drugs or even sunlight.<\/li>\n<li>There is no cure, but treatments can manage the symptoms.<\/li>\n<\/ul>\n        <\/div>\n    <\/div>\n\n    ","protected":false},"excerpt":{"rendered":"<p>RNA-based medicine is about to take a huge leap forward thanks to the discovery of extremely short RNA fragments with important anti-inflammatory properties.&hellip;&nbsp;&nbsp;<a class=\"read-more\" href=\"https:\/\/hudson.org.au\/news\/tiny-rna-fragments-unlock-massive-autoimmune-treatment-potential\/\">Read more<\/a><\/p>\n","protected":false},"featured_media":70576,"menu_order":0,"template":"","issue":[],"tag":[],"story-type":[85],"treatment-type":[94],"class_list":["post-70575","hdsn_news_post","type-hdsn_news_post","status-publish","has-post-thumbnail","hentry","story-type-discovery","treatment-type-immunotherapies"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v27.3 (Yoast SEO v27.3) - 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