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According to an announcement made by Indiana University in July 2024, this new gene therapy has been tested on mice and very successful results have been obtained. As DMD Warrior, we reached out to Dr. Renzhi Han and asked about the current status of this study.<\/p>\n\n\n\n
\u5728\u4ed6\u7d27\u5f20\u7684\u5de5\u4f5c\u4e2d\uff0c\u4ed6\u56de\u7b54\u6211\u4eec\uff1a\u201c\u6211\u4eec\u7684\u65b0\u57fa\u56e0\u6cbb\u7597\u65b9\u6cd5\u5728\u4e34\u5e8a\u524d\u52a8\u7269\u6a21\u578b\u4e2d\u5df2\u663e\u793a\u51fa\u826f\u597d\u7684\u524d\u666f\u3002\u6211\u4eec\u73b0\u5728\u6b63\u5728\u5bfb\u6c42\u8d44\u91d1\uff0c\u4ee5\u4fbf\u5728\u5927\u7ea6\u4e24\u5e74\u540e\u5f00\u59cb\u4e34\u5e8a\u8bd5\u9a8c\u4e4b\u524d\u8fdb\u884c\u66f4\u591a\u7684\u4e34\u5e8a\u524d\u529f\u6548\u548c\u5b89\u5168\u6027\u6d4b\u8bd5\u3002\u201d<\/p>\n\n\n\n
\u201c\u76ee\u524d\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u57fa\u56e0\u7597\u6cd5\u5229\u7528\u7684\u662f\u622a\u77ed\u7248\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff0c\u201d\u8fd9\u9879\u7814\u7a76\u7684\u8d44\u6df1\u4f5c\u8005\u3001\u5370\u7b2c\u5b89\u7eb3\u5927\u5b66\u533b\u5b66\u9662\u513f\u79d1\u6559\u6388 Renzhi Han \u535a\u58eb\u8bf4\u3002\u201c\u4e0d\u5e78\u7684\u662f\uff0c\u8fd9\u79cd\u9009\u62e9\u4e0d\u80fd\u5b8c\u5168\u4fdd\u62a4\u808c\u8089\uff0c\u56e0\u4e3a\u5b83\u7f3a\u4e4f\u5168\u957f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u8bb8\u591a\u91cd\u8981\u529f\u80fd\u57df\u3002\u201d<\/p>\n\n\n\n
\u5c3d\u7ba1\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40\u6700\u8fd1\u6279\u51c6\u4e86\u4e00\u79cd\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u7597\u6cd5\uff0c\u4f46\u97e9\u8868\u793a\u6cbb\u7597\u7ed3\u679c\u5e76\u4e0d\u50cf\u9884\u671f\u7684\u90a3\u6837\u4ee4\u4eba\u6ee1\u610f\u3002<\/p>\n\n\n\n
\u57fa\u4e8e\u4f7f\u7528\u817a\u76f8\u5173\u75c5\u6bd2\u65b9\u6cd5\u5c06\u8d85\u5927\u6cbb\u7597\u57fa\u56e0\u9012\u9001\u81f3\u7ec6\u80de\u7684\u7ecf\u9a8c\uff0cHan \u53ca\u5176\u8d6b\u5c14\u66fc\u00b7B\u00b7\u5a01\u5c14\u65af\u513f\u79d1\u7814\u7a76\u4e2d\u5fc3\u7684\u56e2\u961f\u5f00\u53d1\u4e86\u4e00\u79cd\u4e09\u91cd\u817a\u76f8\u5173\u75c5\u6bd2\u8f7d\u4f53\u7cfb\u7edf\uff0c\u5c06\u5b8c\u6574\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u9012\u9001\u81f3\u808c\u8089\u4e2d\u3002<\/p>\n\n\n\n
“We optimized and tested our new three-vector system to make sure it produced and assembled the full-length dystrophin protein effectively,” Han said. “Our data confirmed we successfully restored full-length dystrophin in both the skeletal and heart muscles of mice with DMD, leading to significant improvements in their muscle health, strength and function.\u201d<\/strong><\/p>\n\n\n\n \u97e9\u5df2\u4e3a\u4ed6\u7684\u4e09\u91cd\u817a\u76f8\u5173\u75c5\u6bd2\u8f7d\u4f53\u7cfb\u7edf\u63d0\u4ea4\u4e86\u4e34\u65f6\u4e13\u5229\u7533\u8bf7\uff0c\u5e76\u6b63\u5728\u4e0e\u5370\u7b2c\u5b89\u7eb3\u5927\u5b66\u521b\u65b0\u548c\u5546\u4e1a\u5316\u529e\u516c\u5ba4\u5408\u4f5c\uff0c\u63a8\u52a8\u8be5\u7597\u6cd5\u7684\u4e0a\u5e02\u3002\u4ed6\u8fd8\u5728\u5bfb\u6c42\u989d\u5916\u8d44\u91d1\uff0c\u4ee5\u4fbf\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u80fd\u591f\u83b7\u5f97\u6709\u5e0c\u671b\u7684\u65b0\u6cbb\u7597\u65b9\u6848\u3002<\/p>\n\n\n\n \u4ed6\u8bf4\u9053\uff1a\u201c\u6211\u76f8\u4fe1\u8fd9\u79cd\u65b0\u7684\u57fa\u56e0\u6cbb\u7597\u65b9\u6cd5\u4e0e\u73b0\u6709\u7684\u6cbb\u7597\u65b9\u6cd5\u76f8\u6bd4\uff0c\u4e3a\u60a3\u8005\u5e26\u6765\u4e86\u663e\u8457\u7684\u4f18\u52bf\uff0c\u6211\u6e34\u671b\u5c06\u5176\u8fdb\u4e00\u6b65\u6295\u5165\u4e34\u5e8a\u5f00\u53d1\u3002\u201d<\/p>\n\n\n\n \u5370\u7b2c\u5b89\u7eb3\u5927\u5b66\u533b\u5b66\u9662\u7684\u5176\u4ed6\u7814\u7a76\u4f5c\u8005\u5305\u62ec\u5468\u8fdc\u3001\u5f20\u6668\u3001\u8096\u4f1f\u4e1c\u548c\u7f57\u5170\u00b7W\u00b7\u8d6b\u4f50\u683c\u3002<\/p>","protected":false},"excerpt":{"rendered":" Dr. Renzhi Han, a professor of pediatrics at Indiana University, is working on a new gene therapy for the treatment of Duchenne (DMD) and is seeking funding for the continuation of the study. According to an announcement made by Indiana University in July 2024, this new gene therapy has been tested on mice and very […]<\/p>\n","protected":false},"author":1,"featured_media":1399,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[359,895,894],"class_list":["post-1398","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-full-length-dystrophin","tag-indiana-university","tag-renzhi-han"],"subtitle":"","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/1398","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/comments?post=1398"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/1398\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/1399"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=1398"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=1398"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=1398"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}