{"id":2231,"date":"2026-09-17T15:21:14","date_gmt":"2026-09-17T15:21:14","guid":{"rendered":"https:\/\/blogs.bmj.com\/jmg\/?p=2231"},"modified":"2026-09-17T15:23:52","modified_gmt":"2026-09-17T15:23:52","slug":"safety-and-efficacy-of-aav-based-mini-and-micro-dystrophin-gene-therapies-in-duchenne-muscular-dystrophy-a-systematic-review-and-meta-analysis-of-clinical-trials-contributed-by-dr-haya-nassour","status":"publish","type":"post","link":"https:\/\/blogs.bmj.com\/jmg\/2026\/09\/17\/safety-and-efficacy-of-aav-based-mini-and-micro-dystrophin-gene-therapies-in-duchenne-muscular-dystrophy-a-systematic-review-and-meta-analysis-of-clinical-trials-contributed-by-dr-haya-nassour\/","title":{"rendered":"Safety and Efficacy of AAV-based Mini- and Micro-Dystrophin Gene Therapies in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis of Clinical Trials (Contributed by Dr Haya Nassour)"},"content":{"rendered":"

Duchenne muscular dystrophy is a genetic disease caused by pathogenic variants in the gene encoding dystrophin, a protein essential for muscle function, leading to progressive muscle weakness and loss of mobility. Several gene replacement therapies that use viral vectors to deliver shortened versions of the dystrophin gene are being tested in clinical trials. We reviewed these studies for safety and efficacy and found modest improvements in motor function, with more consistent benefits in younger children. Most patients experienced mild, transient side effects, although serious complications, including liver injury and rare fatal events, were reported. These findings support the potential of these gene therapies while highlighting the need for careful patient selection, safety monitoring, and longer-term studies. (https:\/\/jmg.bmj.com\/content\/early\/2026\/09\/15\/jmg-2026-111649<\/a>)<\/p>\n","protected":false},"excerpt":{"rendered":"

Duchenne muscular dystrophy is a genetic disease caused by pathogenic variants in the gene encoding dystrophin, a protein essential for muscle function, leading to progressive muscle weakness and loss of mobility. Several gene replacement therapies that use viral vectors to deliver shortened versions of the dystrophin gene are being tested in clinical trials. We reviewed […]<\/p>\n

Read More…<\/a><\/p>\n","protected":false},"author":123,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1],"tags":[],"class_list":["post-2231","post","type-post","status-publish","format-standard","hentry","category-uncategorized"],"yoast_head":"\nSafety and Efficacy of AAV-based Mini- and Micro-Dystrophin Gene Therapies in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis of Clinical Trials (Contributed by Dr Haya Nassour) - JMG Contact 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:\/\/blogs.bmj.com\/jmg\/2026\/09\/17\/safety-and-efficacy-of-aav-based-mini-and-micro-dystrophin-gene-therapies-in-duchenne-muscular-dystrophy-a-systematic-review-and-meta-analysis-of-clinical-trials-contributed-by-dr-haya-nassour\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Safety and Efficacy of AAV-based Mini- and Micro-Dystrophin Gene Therapies in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis of Clinical Trials (Contributed by Dr Haya Nassour) - JMG Contact blog\" \/>\n<meta property=\"og:description\" content=\"Duchenne muscular dystrophy is a genetic disease caused by pathogenic variants in the gene encoding dystrophin, a protein essential for muscle function, leading to progressive muscle weakness and loss of mobility. Several gene replacement therapies that use viral vectors to deliver shortened versions of the dystrophin gene are being tested in clinical trials. We reviewed [...]Read More...\" \/>\n<meta property=\"og:url\" content=\"https:\/\/blogs.bmj.com\/jmg\/2026\/09\/17\/safety-and-efficacy-of-aav-based-mini-and-micro-dystrophin-gene-therapies-in-duchenne-muscular-dystrophy-a-systematic-review-and-meta-analysis-of-clinical-trials-contributed-by-dr-haya-nassour\/\" \/>\n<meta property=\"og:site_name\" content=\"JMG Contact blog\" \/>\n<meta property=\"article:published_time\" content=\"2026-09-17T15:21:14+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2026-09-17T15:23:52+00:00\" \/>\n<meta name=\"author\" content=\"hqqu\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:creator\" content=\"@HuiQiQu\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"hqqu\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"1 minute\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2026\\\/09\\\/17\\\/safety-and-efficacy-of-aav-based-mini-and-micro-dystrophin-gene-therapies-in-duchenne-muscular-dystrophy-a-systematic-review-and-meta-analysis-of-clinical-trials-contributed-by-dr-haya-nassour\\\/#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2026\\\/09\\\/17\\\/safety-and-efficacy-of-aav-based-mini-and-micro-dystrophin-gene-therapies-in-duchenne-muscular-dystrophy-a-systematic-review-and-meta-analysis-of-clinical-trials-contributed-by-dr-haya-nassour\\\/\"},\"author\":{\"name\":\"hqqu\",\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/#\\\/schema\\\/person\\\/be0250f8d5b52412c3e7c222dabd591b\"},\"headline\":\"Safety and Efficacy of AAV-based Mini- and Micro-Dystrophin Gene Therapies in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis of Clinical Trials (Contributed by Dr Haya Nassour)\",\"datePublished\":\"2026-09-17T15:21:14+00:00\",\"dateModified\":\"2026-09-17T15:23:52+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2026\\\/09\\\/17\\\/safety-and-efficacy-of-aav-based-mini-and-micro-dystrophin-gene-therapies-in-duchenne-muscular-dystrophy-a-systematic-review-and-meta-analysis-of-clinical-trials-contributed-by-dr-haya-nassour\\\/\"},\"wordCount\":151,\"commentCount\":0,\"publisher\":{\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/#organization\"},\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"CommentAction\",\"name\":\"Comment\",\"target\":[\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2026\\\/09\\\/17\\\/safety-and-efficacy-of-aav-based-mini-and-micro-dystrophin-gene-therapies-in-duchenne-muscular-dystrophy-a-systematic-review-and-meta-analysis-of-clinical-trials-contributed-by-dr-haya-nassour\\\/#respond\"]}]},{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2026\\\/09\\\/17\\\/safety-and-efficacy-of-aav-based-mini-and-micro-dystrophin-gene-therapies-in-duchenne-muscular-dystrophy-a-systematic-review-and-meta-analysis-of-clinical-trials-contributed-by-dr-haya-nassour\\\/\",\"url\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2026\\\/09\\\/17\\\/safety-and-efficacy-of-aav-based-mini-and-micro-dystrophin-gene-therapies-in-duchenne-muscular-dystrophy-a-systematic-review-and-meta-analysis-of-clinical-trials-contributed-by-dr-haya-nassour\\\/\",\"name\":\"Safety and Efficacy of AAV-based Mini- and Micro-Dystrophin Gene Therapies in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis of Clinical Trials (Contributed by Dr Haya Nassour) - 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