{"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\/ar\/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":"\u064a\u0642\u062f\u0645 Precision BioSciences \u0628\u064a\u0627\u0646\u0627\u062a \u0645\u0627 \u0642\u0628\u0644 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u062d\u0648\u0644 \u0627\u0644\u0641\u0639\u0627\u0644\u064a\u0629 \u0648\u0627\u0644\u0645\u062a\u0627\u0646\u0629 \u0644\u0640 PBGENE-DMD \u0644\u0639\u0644\u0627\u062c \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646 (DMD) \u0641\u064a \u0627\u0644\u0645\u0624\u062a\u0645\u0631 \u0627\u0644\u0633\u0631\u064a\u0631\u064a \u0648\u0627\u0644\u0639\u0644\u0645\u064a \u0644\u062c\u0645\u0639\u064a\u0629 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a (MDA) \u0644\u0639\u0627\u0645 2025"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u0647\u0630\u0627 \u0627\u0644\u0646\u0647\u062c \u0644\u062a\u0635\u062d\u064a\u062d \u062c\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646\u060c \u0648\u0627\u0644\u0630\u064a \u064a\u062a\u0636\u0645\u0646 \u062a\u0639\u062f\u064a\u0644 \u0627\u0644\u062e\u0644\u0627\u064a\u0627 \u0627\u0644\u062c\u0630\u0639\u064a\u0629 \u0627\u0644\u0633\u0627\u062a\u0644\u064a\u0629 \u0627\u0644\u0639\u0636\u0644\u064a\u0629\u060c \u064a\u064f\u062d\u0633\u0651\u0646 \u0628\u0634\u0643\u0644 \u0643\u0628\u064a\u0631 \u0645\u0646 \u0627\u0644\u0645\u062a\u0627\u0646\u0629 \u0648\u0627\u0644\u0646\u062a\u0627\u0626\u062c \u0627\u0644\u0648\u0638\u064a\u0641\u064a\u0629 \u0645\u0642\u0627\u0631\u0646\u0629\u064b \u0628\u0627\u0644\u0637\u0631\u0642 \u0627\u0644\u0627\u0635\u0637\u0646\u0627\u0639\u064a\u0629. \u0648\u0628\u0645\u0627 \u0623\u0646 \u0645\u0627 \u064a\u0635\u0644 \u0625\u0644\u0649 60% \u0645\u0646 \u062d\u0627\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646 \u0646\u0627\u062a\u062c\u0629 \u0639\u0646 \u0639\u064a\u0648\u0628 \u0628\u064a\u0646 \u0627\u0644\u0625\u0643\u0633\u0648\u0646\u0627\u062a 45 \u064855\u060c \u0641\u0625\u0646 \u0647\u0630\u0627 \u0627\u0644\u0646\u0647\u062c \u0623\u0643\u062b\u0631 \u0642\u0627\u0628\u0644\u064a\u0629 \u0644\u0644\u062a\u0637\u0628\u064a\u0642 \u0639\u0644\u0649 \u0646\u0637\u0627\u0642 \u0623\u0648\u0633\u0639 \u0644\u062f\u0649 \u063a\u0627\u0644\u0628\u064a\u0629 \u0645\u0631\u0636\u0649 \u062f\u0648\u0634\u064a\u0646 \u0645\u0642\u0627\u0631\u0646\u0629\u064b \u0628\u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0630\u064a\u0646 \u064a\u0639\u0627\u0646\u0648\u0646 \u0645\u0646 \u062e\u0644\u0644 \u0641\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646\u0627\u062a.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0648\u062a\u062a\u0636\u0645\u0646 \u0627\u0644\u0646\u062a\u0627\u0626\u062c \u0627\u0644\u0631\u0626\u064a\u0633\u064a\u0629 \u0644\u0644\u062f\u0631\u0627\u0633\u0629 \u0645\u0627 \u064a\u0644\u064a:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u0625\u0646\u062a\u0627\u062c \u0628\u0631\u0648\u062a\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0627\u0644\u0648\u0638\u064a\u0641\u064a:<\/strong> \u0646\u062c\u062d PBGENE-DMD \u0641\u064a \u0627\u0633\u062a\u0639\u0627\u062f\u0629 \u0642\u062f\u0631\u0629 \u0627\u0644\u062c\u0633\u0645 \u0639\u0644\u0649 \u0625\u0646\u062a\u0627\u062c \u0628\u0631\u0648\u062a\u064a\u0646 \u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0648\u0638\u064a\u0641\u064a \u0639\u0628\u0631 \u0639\u0636\u0644\u0627\u062a \u0645\u062a\u0639\u062f\u062f\u0629\u060c \u0628\u0645\u0627 \u0641\u064a \u0630\u0644\u0643 \u0639\u0636\u0644\u0627\u062a \u0627\u0644\u0642\u0644\u0628 \u0648\u0627\u0644\u062d\u062c\u0627\u0628 \u0627\u0644\u062d\u0627\u062c\u0632 \u0648\u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u0647\u064a\u0643\u0644\u064a\u0629 \u0628\u0645\u0633\u062a\u0648\u064a\u0627\u062a \u0645\u0646 \u0627\u0644\u0645\u062a\u0648\u0642\u0639 \u0623\u0646 \u062a\u0648\u0641\u0631 \u0641\u0627\u0626\u062f\u0629 \u0639\u0644\u0627\u062c\u064a\u0629.<\/li>\n\n\n\n<li><strong>\u062a\u0639\u0632\u064a\u0632 \u0645\u0631\u0648\u0646\u0629 \u0627\u0644\u0639\u0636\u0644\u0627\u062a:<\/strong> \u0623\u0638\u0647\u0631\u062a \u0627\u0644\u0641\u0626\u0631\u0627\u0646 \u0627\u0644\u0645\u0639\u0627\u0644\u062c\u0629 \u062a\u062d\u0633\u0646\u064b\u0627 \u0641\u064a \u0645\u0642\u0627\u0648\u0645\u0629 \u0627\u0644\u0625\u0635\u0627\u0628\u0629 \u0627\u0644\u0634\u0627\u0630\u0629 \u0628\u0645\u0642\u062f\u0627\u0631 66%\u060c \u0648\u0647\u0648 \u0645\u0624\u0634\u0631 \u0639\u0644\u0649 \u062a\u0639\u0632\u064a\u0632 \u0645\u0631\u0648\u0646\u0629 \u0627\u0644\u0639\u0636\u0644\u0627\u062a\u060c \u0645\u0642\u0627\u0631\u0646\u0629 \u0628\u0646\u0638\u064a\u0631\u0627\u062a\u0647\u0627 \u0627\u0644\u0645\u0631\u064a\u0636\u0629 \u063a\u064a\u0631 \u0627\u0644\u0645\u0639\u0627\u0644\u062c\u0629.<\/li>\n\n\n\n<li><strong>\u0627\u0644\u062a\u062d\u0633\u064a\u0646 \u0627\u0644\u0648\u0638\u064a\u0641\u064a \u0639\u0644\u0649 \u0627\u0644\u0645\u062f\u0649 \u0627\u0644\u0637\u0648\u064a\u0644:<\/strong> \u0641\u064a \u0627\u0644\u0641\u0626\u0631\u0627\u0646 \u0627\u0644\u0645\u0639\u0627\u0644\u062c\u0629 \u0628\u0640 PBGENE-DMD\u060c \u0648\u0635\u0644 \u0627\u0644\u062d\u062f \u0627\u0644\u0623\u0642\u0635\u0649 \u0644\u0642\u0648\u0629 \u0627\u0644\u0625\u062e\u0631\u0627\u062c (MFO)\u060c \u0648\u0647\u0648 \u0645\u0642\u064a\u0627\u0633 \u0648\u0638\u064a\u0641\u064a \u062d\u0627\u0633\u0645\u060c \u0625\u0644\u0649 93% \u0645\u0646 MFO \u0641\u064a \u0627\u0644\u0641\u0626\u0631\u0627\u0646 \u0627\u0644\u0633\u0644\u064a\u0645\u0629 \u0645\u0639 \u0645\u0644\u0627\u062d\u0638\u0629 \u0627\u0644\u062a\u062d\u0633\u0646 \u0641\u064a \u0627\u0644\u0641\u0626\u0631\u0627\u0646 \u0627\u0644\u0645\u0639\u0627\u0644\u062c\u0629 \u0628\u0640 PBGENE-DMD \u0628\u064a\u0646 3 \u06486 \u0623\u0634\u0647\u0631.<\/li>\n\n\n\n<li><strong>\u0646\u062a\u0627\u0626\u062c \u062f\u0627\u0626\u0645\u0629:<\/strong> \u062a\u0645 \u0627\u0644\u0643\u0634\u0641 \u0639\u0646 \u0646\u0633\u062e\u0629 mRNA \u0627\u0644\u0645\u0639\u062f\u0644\u0629 \u0628\u0648\u0627\u0633\u0637\u0629 PBGENE-DMD \u0644\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0641\u064a \u062e\u0644\u0627\u064a\u0627 PAX7+\u060c \u0648\u0647\u064a \u0639\u0644\u0627\u0645\u0629 \u0644\u062e\u0644\u0627\u064a\u0627 \u062c\u0630\u0639\u064a\u0629 \u0642\u0645\u0631\u064a\u0629 \u0644\u0644\u0639\u0636\u0644\u0627\u062a\u060c \u0645\u0645\u0627 \u064a\u0634\u064a\u0631 \u0625\u0644\u0649 \u0625\u0645\u0643\u0627\u0646\u064a\u0629 \u062a\u062d\u0642\u064a\u0642 \u062a\u0623\u062b\u064a\u0631\u0627\u062a \u0639\u0644\u0627\u062c\u064a\u0629 \u062f\u0627\u0626\u0645\u0629 \u0645\u0642\u0627\u0631\u0646\u0629 \u0628\u0623\u0633\u0627\u0644\u064a\u0628 \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a \u0627\u0644\u0642\u064a\u0627\u0633\u064a\u0629.<\/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"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/3288","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/comments?post=3288"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/3288\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media\/3289"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media?parent=3288"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/categories?post=3288"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/tags?post=3288"}],"curies":[{"name":"\u0648\u0648\u0631\u062f\u0628\u0631\u064a\u0633","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}