{"id":14826,"date":"2025-09-26T17:00:00","date_gmt":"2025-09-26T15:00:00","guid":{"rendered":"https:\/\/holistic.news\/en\/?p=14826"},"modified":"2025-10-20T09:04:30","modified_gmt":"2025-10-20T07:04:30","slug":"hope-after-a-dna-error-muscle-dysfunction-treatment","status":"publish","type":"post","link":"https:\/\/holistic.news\/en\/hope-after-a-dna-error-muscle-dysfunction-treatment\/","title":{"rendered":"Hope after a DNA error. Muscle dysfunction treatment."},"content":{"rendered":"\n<h2 class=\"wp-block-heading\">Closer to a cure for an \u201cincurable\u201d disease<\/h2>\n\n\n\n<p><strong>Multisystem smooth muscle dysfunction syndrome (MSMDS)<\/strong> is a sentence for parents. The disease leads to <strong>strokes, aortic dissection and, in many cases, simply death in childhood.<\/strong> It stems from a single error in the <strong>ACTA2<\/strong> gene, which encodes smooth muscle actin. Until now, MSMDS had no cure. But the latest experiment, reported in <strong><a href=\"https:\/\/www.nature.com\/articles\/s41551-025-01499-1\" target=\"_blank\" rel=\"noreferrer noopener nofollow\">Nature Biomedical Engineering<\/a><\/strong>, opens a new chapter in the search for an effective way to treat smooth muscle dysfunction.<\/p>\n\n\n\n<p><em>\u201cThe story of this research really began at the patient\u2019s bedside,\u201d<\/em> says <strong>Dr. Patricia Musolino<\/strong> of Massachusetts General Hospital. <em>\u201cAn infant in critical condition first brought our team together \u2014 spanning clinical, genetic, biological and therapeutic expertise in this disease,\u201d<\/em> she adds. So what exactly is the method the research team developed?<\/p>\n\n\n\n<p><strong>You might like to read:<\/strong> <a href=\"https:\/\/holistic.news\/en\/viruses-in-our-dna\/\" target=\"_blank\" rel=\"noreferrer noopener\">Viruses in our DNA: can we switch them off?<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Toward more effective muscle dysfunction treatment<\/h2>\n\n\n\n<p>Can we reprogram nature? Physicians at Mass General Brigham set out to try. They used <strong>CRISPR<\/strong> technology re-engineered for MSMDS. It relies on binding <strong>CRISPR-Cas9<\/strong> and a dedicated enzyme to a specific DNA strand and making a cut. The first problem? Standard CRISPR tools,<strong> while correcting the mutation, also damaged nearby regions of the <a href=\"https:\/\/holistic.news\/en\/genetic-basis-of-dyslexia-found\/\">genome<\/a><\/strong>. That flaw had to be eliminated.<\/p>\n\n\n\n<p>The team led by <strong>Dr. Benjamin Kleinstiver<\/strong> tested dozens of system variants until they created a finely tuned <strong>base editor<\/strong> \u2014 a protein tool that modulates the genome with high specificity. The result? <strong>One injection extended the lifespan of MSMDS mice by 4 times.<\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">From lab bench to hospital<\/h2>\n\n\n\n<p><em>\u201cOur laboratory has advanced the design of base editors to make them safer, more effective and more precise \u2014 and therefore better suited to treating genetic disease,\u201d <\/em>Kleinstiver told Medical Xpress. The biggest challenge, however, was delivering the therapy to the right place. <strong>MSMDS targets blood vessels, so the treatment has to go exactly there<\/strong>. <strong>Dr. Casey Maguire <\/strong>designed a specialised viral vector that homes to the smooth muscle lining of vessels.<\/p>\n\n\n\n<p>The researchers are already in talks with the <strong>U.S. Food and Drug Administration (FDA)<\/strong> \u2014 a first step toward human clinical trials. And this is only the beginning. The same technology could aid in treating other conditions: <strong>Marfan syndrome<\/strong>, <strong>moyamoya disease<\/strong>, and even <strong>atherosclerosis<\/strong> \u2014 the world\u2019s leading cause of death.<\/p>\n\n\n\n<p><em>\u201cThe impact of this work extends beyond a single disease,\u201d<\/em> says <strong>Dr. Mark Lindsay<\/strong> of the Mass General Brigham Heart and Vascular Institute. \u201c<em>Our team has built tools that have accelerated the science of genome editing and precision therapies to levels that were unthinkable just two years ago.\u201d<\/em> Are we witnessing the dawn of a new era in medicine \u2014 one where rewriting the genetic code becomes as routine as writing a prescription \u2014 and where muscle dysfunction treatment moves from hope to standard care?<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p><em>Read this article in Polish<\/em>: <a href=\"https:\/\/holistic.news\/leczenie-dysfunkcji-miesni-edycja-genow\/\" target=\"_blank\" rel=\"noreferrer noopener\">Dzieci umiera\u0142y przez&nbsp;b\u0142\u0105d w&nbsp;DNA. Teraz pojawi\u0142a si\u0119 nadzieja<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>A single letter in DNA can determine a child\u2019s life. Scientists in Boston have shown it can be corrected \u2014 and early tests extended animal lifespan 4 times. This could be the start of a muscle dysfunction treatment that saves the youngest patients now condemned to a fatal disease.<\/p>\n","protected":false},"author":275,"featured_media":14829,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[435],"tags":[1331,1439,1833,1179,1097,1832,1834],"class_list":["post-14826","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-science","tag-crispr-cas9","tag-dna","tag-gene","tag-health","tag-medical-research","tag-muscle-dysfunction-treatment","tag-treatment"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v27.4 (Yoast SEO v27.4) - https:\/\/yoast.com\/product\/yoast-seo-premium-wordpress\/ -->\n<title>Hope after a DNA error. 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See what Boston clinicians have achieved with a base-editing approach for MSMDS.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/holistic.news\/en\/hope-after-a-dna-error-muscle-dysfunction-treatment\/\" \/>\n<meta property=\"og:site_name\" content=\"Holistic News\" \/>\n<meta property=\"article:publisher\" content=\"https:\/\/www.facebook.com\/portalHolisticNews\/\" \/>\n<meta property=\"article:published_time\" content=\"2025-09-26T15:00:00+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2025-10-20T07:04:30+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/holistic.news\/en\/wp-content\/uploads\/2025\/09\/Muscle-dysfunction-treatment.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"1200\" \/>\n\t<meta property=\"og:image:height\" content=\"675\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"author\" content=\"Maciej Bartusik\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:creator\" content=\"@news_holistic\" \/>\n<meta name=\"twitter:site\" content=\"@news_holistic\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"Maciej Bartusik\" \/>\n\t<meta name=\"twitter:label2\" content=\"Estimated reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"3 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\\\/\\\/holistic.news\\\/en\\\/hope-after-a-dna-error-muscle-dysfunction-treatment\\\/#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/holistic.news\\\/en\\\/hope-after-a-dna-error-muscle-dysfunction-treatment\\\/\"},\"author\":{\"name\":\"Maciej Bartusik\",\"@id\":\"https:\\\/\\\/holistic.news\\\/en\\\/#\\\/schema\\\/person\\\/0758fb0cf338b71ce51e2da415bc266c\"},\"headline\":\"Hope after a DNA error. 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