{"id":3288,"date":"2025-03-19T16:03:20","date_gmt":"2025-03-19T13:03:20","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3288"},"modified":"2025-05-15T14:31:04","modified_gmt":"2025-05-15T11:31:04","slug":"precision-biosciences-presents-preclinical-efficacy-and-durability-data-on-pbgene-dmd-for-the-treatment-of-duchenne-muscular-dystrophy-dmd-at-the-2025-muscular-dystrophy-association-mda-clinical","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/precision-biosciences-presents-preclinical-efficacy-and-durability-data-on-pbgene-dmd-for-the-treatment-of-duchenne-muscular-dystrophy-dmd-at-the-2025-muscular-dystrophy-association-mda-clinical\/","title":{"rendered":"Precision BioSciences pr\u00e9sente des donn\u00e9es pr\u00e9cliniques sur l&#039;efficacit\u00e9 et la durabilit\u00e9 du PBGENE-DMD pour le traitement de la dystrophie musculaire de Duchenne (DMD) lors de la conf\u00e9rence clinique et scientifique 2025 de la Muscular Dystrophy Association (MDA)"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Cette approche de correction du g\u00e8ne de la dystrophine, qui consiste \u00e0 modifier les cellules souches satellites musculaires, am\u00e9liore potentiellement la durabilit\u00e9 et les r\u00e9sultats fonctionnels par rapport aux approches synth\u00e9tiques. \u00c9tant donn\u00e9 que jusqu&#039;\u00e0 60% des cas de DMD sont caus\u00e9s par des d\u00e9fauts entre les exons 45 et 55, cette approche est plus largement applicable \u00e0 la majorit\u00e9 des patients atteints de DMD que les cas de saut d&#039;exon.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Les principales conclusions de l\u2019\u00e9tude sont les suivantes :<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Production de prot\u00e9ines dystrophines fonctionnelles :<\/strong> Le PBGENE-DMD a restaur\u00e9 la capacit\u00e9 du corps \u00e0 produire une prot\u00e9ine dystrophine fonctionnelle dans plusieurs muscles, notamment le c\u0153ur, le diaphragme et les muscles squelettiques, \u00e0 des niveaux cens\u00e9s apporter un b\u00e9n\u00e9fice th\u00e9rapeutique.<\/li>\n\n\n\n<li><strong>R\u00e9silience musculaire am\u00e9lior\u00e9e :<\/strong> Les souris trait\u00e9es ont montr\u00e9 une am\u00e9lioration de 66% de la r\u00e9sistance aux blessures excentriques, un indicateur d&#039;une r\u00e9silience musculaire am\u00e9lior\u00e9e, par rapport \u00e0 leurs homologues malades non trait\u00e9es.<\/li>\n\n\n\n<li><strong>Am\u00e9lioration fonctionnelle \u00e0 long terme :<\/strong> Chez les souris trait\u00e9es avec PBGENE-DMD, la force de sortie maximale (MFO), une mesure fonctionnelle critique, a atteint jusqu&#039;\u00e0 93% de la MFO chez les souris t\u00e9moins saines avec une am\u00e9lioration observ\u00e9e chez les souris trait\u00e9es avec PBGENE-DMD entre 3 et 6 mois.<\/li>\n\n\n\n<li><strong>R\u00e9sultats durables :<\/strong> Le transcrit d&#039;ARNm de la dystrophine \u00e9dit\u00e9 par PBGENE-DMD a \u00e9t\u00e9 d\u00e9tect\u00e9 dans les cellules PAX7+, un marqueur des cellules souches satellites musculaires, sugg\u00e9rant un potentiel d&#039;effets th\u00e9rapeutiques durables par rapport aux approches de th\u00e9rapie g\u00e9nique standard.<\/li>\n<\/ul>","protected":false},"excerpt":{"rendered":"<p>This dystrophin gene correction approach which involves editing muscle satellite stem cells potentially enhances durability and functional outcomes compared to synthetic approaches. Since up to 60% of DMD cases are caused by defects between exons 45 and 55, this approach is more broadly applicable for the majority of DMD patients than exon skippers. Key findings [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3289,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[359,72,297,314,313],"class_list":["post-3288","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-full-length-dystrophin","tag-gene-editing","tag-muscular-dystrophy-association","tag-pbgene-dmd","tag-precision-biosciences"],"subtitle":"\u00c9tude d'\u00e9dition g\u00e9n\u00e9tique Precision BioSciences PBGENE-DMD : en supprimant pr\u00e9cis\u00e9ment et directement la cause g\u00e9n\u00e9tique fondamentale des patients atteints de DMD pr\u00e9sentant des d\u00e9fauts entre l'exon 45 et 55, l'approche de la soci\u00e9t\u00e9 pourrait fournir des r\u00e9sultats plus durables pour ces patients par rapport aux th\u00e9rapies g\u00e9niques \u00e0 base de microdystrophine.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/3288","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/comments?post=3288"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/3288\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/3289"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=3288"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=3288"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=3288"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}