{"id":1134,"date":"2014-01-14T18:01:58","date_gmt":"2014-01-14T17:01:58","guid":{"rendered":"https:\/\/www.aica3.org\/cms\/?p=1134"},"modified":"2022-01-06T09:56:56","modified_gmt":"2022-01-06T08:56:56","slug":"sicurezza-ed-efficacia-del-trasferimento-genico-aav-mediato-di-calpaina-3-in-un-modello-murino-di-distrofia-muscolare-dei-cingoli-tipo-2a","status":"publish","type":"post","link":"https:\/\/www.aica3.org\/cms\/en\/sicurezza-ed-efficacia-del-trasferimento-genico-aav-mediato-di-calpaina-3-in-un-modello-murino-di-distrofia-muscolare-dei-cingoli-tipo-2a\/","title":{"rendered":"Safety and Efficacy of AAV-Mediated Calpain 3Gene Transfer in a Mouse Model of Limb-Girdle Muscular Dystrophy Type 2A"},"content":{"rendered":"<p><em>Marc Bartoli, Carinne Roudaut, Samia Martin, Franc\u00b8oise Fougerousse, Laurence Suel,<br \/>\nJe\u00b4ro\u02c6me Poupiot, Evelyne Gicquel, Fanny Noulet, Olivier Danos, and Isabelle Richard<br \/>\nMol Ther. 2006 Feb;13(2):250-9. Epub 2005 Nov 14<\/em><\/p>\n<p class=\"translation-block\"><strong>Safety and Efficacy of AAV-Mediated Calpain 3Gene Transfer in a Mouse Model of Limb-Girdle Muscular Dystrophy Type 2A<\/strong><br>\nCalpainopathy (limb-girdle muscular dystrophy type 2A, LGMD2A) is a recessive muscular disorder caused by deficiency in the calcium-dependent cysteine protease calpain 3. To date, no treatment exists for this disease. We evaluated the potential of recombinant adeno-associated virus (rAAV)vectors for gene therapy in a murine model for LGMD2A. To drive the expression of calpain 3, weused rAAV2\/1 pseudotyped vectors and muscle-specific promoters to avoid calpain 3 cell toxicity. We report efficient and stable transgene expression in muscle with restoration of the proteolytic activity and without evident toxicity. In addition, calpain 3 was correctly targeted to the sarcomere. Moreover, its presence resulted in improvement of the histological features and in therapeuticefficacy at the physiological levels, including correction of atrophy and full rescue of the contractile force deficits. Our results establish the feasibility of AAV-mediated calpain 3 gene transfer as a\ntherapeutic approach.<\/p>\n<p><a href=\"https:\/\/www.aica3.org\/cms\/wp-content\/uploads\/2014\/01\/1.bartoli2006.pdf\" target=\"_blank\" rel=\"noopener\">Click to download the full version of the article<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Marc Bartoli, Carinne Roudaut, Samia Martin, Franc\u00b8oise Fougerousse, Laurence Suel, Je\u00b4ro\u02c6me Poupiot, Evelyne Gicquel, Fanny Noulet, Olivier Danos, and Isabelle Richard Mol Ther. 2006 Feb;13(2):250-9. Epub 2005 Nov 14 Sicurezza ed efficacia del trasferimento genico AAV-mediato di Calpaina 3 in un modello murino di distrofia muscolare dei cingoli tipo 2A La distrofia muscolare dei cingoli [&hellip;]<\/p>\n","protected":false},"author":4,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_et_pb_use_builder":"","_et_pb_old_content":"","_et_gb_content_width":"","footnotes":""},"categories":[34,33],"tags":[],"class_list":["post-1134","post","type-post","status-publish","format-standard","hentry","category-articoli-scientifici","category-ricerca-e-pubblicazioni"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Sicurezza ed efficacia del trasferimento genico AAV-mediato di Calpaina 3 in un modello murino di distrofia muscolare dei cingoli tipo 2A - AICa3 - ETS - Associazione Italiana Calpaina 3<\/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:\/\/www.aica3.org\/cms\/en\/sicurezza-ed-efficacia-del-trasferimento-genico-aav-mediato-di-calpaina-3-in-un-modello-murino-di-distrofia-muscolare-dei-cingoli-tipo-2a\/\" \/>\n<meta property=\"og:locale\" content=\"en_GB\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Sicurezza ed efficacia del trasferimento genico AAV-mediato di Calpaina 3 in un modello murino di distrofia muscolare dei cingoli tipo 2A - AICa3 - ETS - Associazione Italiana Calpaina 3\" \/>\n<meta property=\"og:description\" content=\"Marc Bartoli, Carinne Roudaut, Samia Martin, Franc\u00b8oise Fougerousse, Laurence Suel, Je\u00b4ro\u02c6me Poupiot, Evelyne Gicquel, Fanny Noulet, Olivier Danos, and Isabelle Richard Mol Ther. 2006 Feb;13(2):250-9. Epub 2005 Nov 14 Sicurezza ed efficacia del trasferimento genico AAV-mediato di Calpaina 3 in un modello murino di distrofia muscolare dei cingoli tipo 2A La distrofia muscolare dei cingoli [&hellip;]\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.aica3.org\/cms\/en\/sicurezza-ed-efficacia-del-trasferimento-genico-aav-mediato-di-calpaina-3-in-un-modello-murino-di-distrofia-muscolare-dei-cingoli-tipo-2a\/\" \/>\n<meta property=\"og:site_name\" content=\"AICa3 - ETS - Associazione Italiana Calpaina 3\" \/>\n<meta property=\"article:publisher\" content=\"http:\/\/www.facebook.com\/pages\/AICa3-Associazione-Italiana-Calpaina-3\/119599211561350\" \/>\n<meta property=\"article:published_time\" content=\"2014-01-14T17:01:58+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2022-01-06T08:56:56+00:00\" \/>\n<meta name=\"author\" content=\"admin\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"admin\" \/>\n\t<meta name=\"twitter:label2\" content=\"Estimated 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:\\\/\\\/www.aica3.org\\\/cms\\\/sicurezza-ed-efficacia-del-trasferimento-genico-aav-mediato-di-calpaina-3-in-un-modello-murino-di-distrofia-muscolare-dei-cingoli-tipo-2a\\\/#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/www.aica3.org\\\/cms\\\/sicurezza-ed-efficacia-del-trasferimento-genico-aav-mediato-di-calpaina-3-in-un-modello-murino-di-distrofia-muscolare-dei-cingoli-tipo-2a\\\/\"},\"author\":{\"name\":\"admin\",\"@id\":\"https:\\\/\\\/www.aica3.org\\\/cms\\\/#\\\/schema\\\/person\\\/8a5e19b3ab1d1aa2190043f3ddb89aa2\"},\"headline\":\"Sicurezza ed efficacia del trasferimento genico AAV-mediato di Calpaina 3 in un modello murino di distrofia muscolare dei cingoli tipo 2A\",\"datePublished\":\"2014-01-14T17:01:58+00:00\",\"dateModified\":\"2022-01-06T08:56:56+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\\\/\\\/www.aica3.org\\\/cms\\\/sicurezza-ed-efficacia-del-trasferimento-genico-aav-mediato-di-calpaina-3-in-un-modello-murino-di-distrofia-muscolare-dei-cingoli-tipo-2a\\\/\"},\"wordCount\":236,\"articleSection\":[\"Articoli scientifici\",\"Ricerca e Pubblicazioni\"],\"inLanguage\":\"en-GB\"},{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/www.aica3.org\\\/cms\\\/sicurezza-ed-efficacia-del-trasferimento-genico-aav-mediato-di-calpaina-3-in-un-modello-murino-di-distrofia-muscolare-dei-cingoli-tipo-2a\\\/\",\"url\":\"https:\\\/\\\/www.aica3.org\\\/cms\\\/sicurezza-ed-efficacia-del-trasferimento-genico-aav-mediato-di-calpaina-3-in-un-modello-murino-di-distrofia-muscolare-dei-cingoli-tipo-2a\\\/\",\"name\":\"Sicurezza ed efficacia del trasferimento genico AAV-mediato di Calpaina 3 in un modello murino di distrofia muscolare dei cingoli tipo 2A - 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