{"id":3860,"date":"2025-05-15T13:01:14","date_gmt":"2025-05-15T10:01:14","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3860"},"modified":"2025-05-15T13:01:16","modified_gmt":"2025-05-15T10:01:16","slug":"precision-biosciences-pbgene-dmd-restores-production-of-nearly-full-length-dystrophin-protein","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/es\/precision-biosciences-pbgene-dmd-restores-production-of-nearly-full-length-dystrophin-protein\/","title":{"rendered":"Precision BioSciences PBGENE-DMD restaura la producci\u00f3n de la prote\u00edna distrofina de longitud casi completa"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Precision BioSciences, anunci\u00f3 hoy la priorizaci\u00f3n estrat\u00e9gica y la aceleraci\u00f3n de PBGENE-DMD, el primer enfoque de edici\u00f3n gen\u00e9tica in vivo de su clase de la compa\u00f1\u00eda para la distrofia muscular de Duchenne (DMD).<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>Tabla de contenido<\/h2><nav><ul><li><a href=\"#what-is-pbgene-dmd\">\u00bfQu\u00e9 es PBGENE-DMD?<\/a><\/li><li><a href=\"#how-the-pbgene-dmd-approach-works\">\u00bfC\u00f3mo funciona el m\u00e9todo PBGENE-DMD?<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-pbgene-dmd\">\u00bfQu\u00e9 es PBGENE-DMD?<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>PBGENE-DMD es un enfoque de edici\u00f3n gen\u00e9tica in vivo de primera clase para la mayor\u00eda de los pacientes con distrofia muscular de Duchenne afectados por mutaciones de distrofina en la regi\u00f3n de &quot;punto caliente&quot; m\u00e1s com\u00fan entre los exones 45-55.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>El candidato cl\u00ednico final PBGENE-DMD demuestra datos precl\u00ednicos convincentes para mejorar de forma duradera el beneficio funcional a lo largo del tiempo.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Objetivo de precisi\u00f3n para presentar una solicitud de nuevo f\u00e1rmaco en investigaci\u00f3n (IND) y\/o de ensayo cl\u00ednico (CTA) para PBGENE-DMD en 2025, con datos cl\u00ednicos esperados para 2026.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Leer m\u00e1s: <a href=\"https:\/\/dmdwarrior.com\/es\/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\/\" target=\"_blank\" rel=\"noreferrer noopener\">PBGENE-DMD para el tratamiento de la distrofia muscular de Duchenne (DMD)<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"how-the-pbgene-dmd-approach-works\">\u00bfC\u00f3mo funciona el m\u00e9todo PBGENE-DMD?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Actualmente no existen terapias aprobadas que puedan generar mejoras funcionales musculares significativas y duraderas. PBGENE-DMD emplea dos nucleasas ARCUS complementarias administradas en un \u00fanico AAV para extirpar los exones 45-55 del gen de la distrofina con el objetivo de restaurar la producci\u00f3n natural del cuerpo de una prote\u00edna distrofina funcional.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">En datos precl\u00ednicos presentados en el ASGCT, PBGENE-DMD demostr\u00f3 una mejora funcional significativa y duradera en un modelo murino humanizado de DMD. Tras la administraci\u00f3n de AAV, PBGENE-DMD restableci\u00f3 la capacidad del organismo para producir distrofina funcional en m\u00faltiples m\u00fasculos, incluyendo el card\u00edaco y el esquel\u00e9tico. A lo largo de 9 meses, los ratones tratados con PBGENE-DMD mostraron un aumento en la expresi\u00f3n de distrofina, lo que result\u00f3 en una mejora funcional muscular sustancial y sostenida. Adem\u00e1s, la transcripci\u00f3n del ARNm de distrofina editado por PBGENE-DMD en c\u00e9lulas madre sat\u00e9lite musculares, que son c\u00e9lulas progenitoras de nuevas c\u00e9lulas musculares, respalda la posibilidad de una durabilidad a largo plazo.<\/p>","protected":false},"excerpt":{"rendered":"<p>Precision BioSciences, today announced the strategic prioritization and acceleration of PBGENE-DMD, the Company\u2019s first-in-class in vivo gene editing approach for Duchenne Muscular Dystrophy (DMD). What is PBGENE-DMD? Read more: PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy (DMD) How the PBGENE-DMD Approach Works? There are currently no approved therapies that can drive significant and durable [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3863,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[359,72,314,313],"class_list":["post-3860","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-full-length-dystrophin","tag-gene-editing","tag-pbgene-dmd","tag-precision-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/posts\/3860","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/comments?post=3860"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/posts\/3860\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/media\/3863"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/media?parent=3860"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/categories?post=3860"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/tags?post=3860"}],"curies":[{"name":"gracias","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}