{"id":1562,"date":"2025-03-12T11:31:49","date_gmt":"2025-03-12T10:31:49","guid":{"rendered":"https:\/\/remeds.org\/?p=1562"},"modified":"2025-05-20T23:55:36","modified_gmt":"2025-05-20T21:55:36","slug":"recruitment-starts-for-hero-crispr-cas13-clinical-trial-for-mds","status":"publish","type":"post","link":"https:\/\/remeds.org\/ja\/2025\/03\/12\/recruitment-starts-for-hero-crispr-cas13-clinical-trial-for-mds\/","title":{"rendered":"MDS\u3092\u5bfe\u8c61\u3068\u3057\u305fCRISPR cas13\u81e8\u5e8a\u8a66\u9a13\u300cHERO\u300d\u306e\u88ab\u9a13\u8005\u52df\u96c6\u958b\u59cb"},"content":{"rendered":"<p><a href=\"https:\/\/dupmecp2.eu\/crispr-cas13\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u6280\u8853\u306b\u3064\u3044\u3066<\/a><\/p>\n\n\n\n<p><strong>\u81e8\u5e8a\u8a66\u9a13\u306e\u6982\u8981<\/strong>&nbsp;<\/p>\n\n\n\n<p>HERO \u8a66\u9a13\u3067\u306f\u3001HG204 \u3092\u8133\u5185\u306b\u76f4\u63a5\u5c4a\u3051\u308b\u305f\u3081\u306b\u8133\u5ba4\u5185\u6295\u4e0e\uff08ICV\uff09\u306b\u3088\u308b\u5358\u56de\u6ce8\u5c04\u304c\u884c\u308f\u308c\u307e\u3059\u30022024 \u5e74 10 \u6708\u306b\u52df\u96c6\u304c\u59cb\u307e\u308a\u3001\u5bfe\u8c61\u306f MECP2 \u91cd\u8907\u75c7\u5019\u7fa4\uff08MDS\uff09\u3068\u8a3a\u65ad\u3055\u308c\u305f 2\uff5e18 \u6b73\u306e\u7537\u6027\u306e\u60a3\u8005\u3055\u3093 6 \u540d\u3067\u3059\u3002\u88ab\u9a13\u8005\u306f\u3001 60 \u9031\u9593\u306b\u308f\u305f\u308b\u8a66\u9a13\u306b\u53c2\u52a0\u3057\u30018 \u9031\u9593\u306e\u30b9\u30af\u30ea\u30fc\u30cb\u30f3\u30b0\u671f\u9593\u3001\u6cbb\u7642\u306e\u5b9f\u65bd\u3001\u305d\u3057\u3066 52 \u9031\u9593\u306e\u30d5\u30a9\u30ed\u30fc\u30a2\u30c3\u30d7\u671f\u9593\u3092\u7d4c\u307e\u3059\u3002\u4e3b\u306a\u76ee\u7684\u306f\u3001\u6709\u5bb3\u4e8b\u8c61\u3092\u76e3\u8996\u3057\u3001\u6cbb\u7642\u5f8c\u306e\u767a\u9054\u306e\u9032\u884c\u3092\u8a55\u4fa1\u3059\u308b\u3053\u3068\u306b\u3042\u308a\u307e\u3059\u3002\u73fe\u5728\u3001\u3053\u306e\u8a66\u9a13\u306f\u4e2d\u56fd\u30fb\u5317\u4eac\u5927\u5b66\u75c5\u9662\u3067\u5b9f\u65bd\u3055\u308c\u3066\u3044\u3066\u3001\u4eca\u5f8c\u306e\u4e2d\u56fd\u3067\u306e\u8ffd\u52a0\u52df\u96c6\u306f\u3001\u6700\u521d\u306b\u6cbb\u7642\u3092\u53d7\u3051\u305f\u60a3\u8005\u306e\u7d50\u679c\u306b\u57fa\u3065\u3044\u3066\u5224\u65ad\u3055\u308c\u307e\u3059\u3002&nbsp;<\/p>\n\n\n\n<p>\u3088\u308a\u8a73\u7d30\u306a\u60c5\u5831\u306b\u3064\u3044\u3066\u306f\u3001\u8a66\u9a13\u5b9f\u65bd\u8a08\u753b\u66f8\u3092\u304a\u8aad\u307f\u3044\u305f\u3060\u304d\u305f\u3044\u3002<\/p>\n\n\n\n<p><a href=\"https:\/\/clinicaltrials.gov\/study\/NCT06615206?cond=Mecp2%20Duplication%20Syndrome&amp;rank=1\" target=\"_blank\" rel=\"noreferrer noopener\">\u7814\u7a76\u8a08\u753b\u66f8\u3078<\/a><\/p>\n\n\n\n<p><strong>\u6700\u521d\u306e\u60a3\u8005\u3078\u306e\u6295\u4e0e\u3068\u4e88\u5099\u7684\u77e5\u898b<\/strong>&nbsp;<\/p>\n\n\n\n<p>11\u6708\u3001HuidaGene\u793e\u306f\u6700\u521d\u306e\u60a3\u8005\u3078\u306eHG204\u306e\u6295\u4e0e\u306b\u6210\u529f\u3057\u305f\u3068\u767a\u8868\u3057\u307e\u3057\u305f\u3002\u6ce8\u5c04\u304b\u30896\u9031\u9593\u5f8c\u3001\u3053\u306e\u60a3\u8005\u306b\u306f\u5927\u304d\u306a\u526f\u4f5c\u7528\u306f\u898b\u3089\u308c\u305a\u3001\u6709\u52b9\u6027\u306e\u4e88\u5099\u7684\u5146\u5019\u304c\u89b3\u5bdf\u3055\u308c\u307e\u3057\u305f\u3002\u4eca\u5f8c\u6570\u9031\u9593\u306b\u308f\u305f\u308a\u3001\u60a3\u8005\u306e\u72b6\u614b\u3092\u6ce8\u610f\u6df1\u304f\u89b3\u5bdf\u3057\u3001\u6cbb\u7642\u306e\u5b89\u5168\u6027\u3068\u6709\u52b9\u6027\u3092\u3055\u3089\u306b\u8a55\u4fa1\u3057\u307e\u3059\u3002&nbsp;<\/p>\n\n\n\n<p><strong>\u6b21\u306e\u30b9\u30c6\u30c3\u30d7\u3068\u76ee\u6a19<\/strong>&nbsp;<\/p>\n\n\n\n<p>HuidaGene\u793e\u306f\u898f\u5236\u5f53\u5c40\u304b\u3089\u5927\u304d\u306a\u524d\u9032\u3092\u9042\u3052\u3001MECP2\u91cd\u8907\u75c7\u5019\u7fa4\u6cbb\u7642\u85ac\u3067\u3042\u308bHG204\u306b\u5bfe\u3057\u3066\u3001\u7c73\u56fdFDA\u304b\u3089\u300c\u5e0c\u5c11\u5c0f\u5150\u75be\u60a3\u6307\u5b9a\uff08RPDD\uff09\u300d\u304a\u3088\u3073\u300c\u30aa\u30fc\u30d5\u30a1\u30f3\u30c9\u30e9\u30c3\u30b0\u6307\u5b9a\uff08ODD\uff09\u300d\u306e2\u3064\u306e\u91cd\u8981\u306a\u6307\u5b9a\u3092\u53d6\u5f97\u3057\u307e\u3057\u305f\u3002HERO\u8a66\u9a13\u306f\u4e2d\u56fd\u3067\u5b9f\u65bd\u3055\u308c\u3066\u3044\u307e\u3059\u304c\u3001\u540c\u793e\u306f\u7c73\u56fdFDA\u3084\u6b27\u5ddeEMA\u3068\u4eca\u5f8c\u306e\u7c73\u56fd\u304a\u3088\u3073\u30e8\u30fc\u30ed\u30c3\u30d1\u3067\u306e\u8ffd\u52a0\u7814\u7a76\u306b\u3064\u3044\u3066\u3082\u5354\u8b70\u3092\u958b\u59cb\u3057\u3066\u3044\u307e\u3059\u3002&nbsp;<\/p>\n\n\n\n<p><strong>\u7814\u7a76\u767a\u8868<\/strong>&nbsp;<\/p>\n\n\n\n<p class=\"translation-block\">\u4e00\u65b9\u3001HuidaGene\u793e\u306f\u3001CRISPR-Cas13\u6280\u8853\u3092\u7528\u3044\u305f\u524d\u81e8\u5e8a\u5b9f\u9a13\u306e\u8a73\u7d30\u3092\u300e<em>Nature Neuroscience<\/em>\u300f\u306b\u767a\u8868\u3057\u307e\u3057\u305f\u3002\u7814\u7a76\u3067\u306f\u3001HG204\u304cMECP2\u306emRNA\u30ec\u30d9\u30eb\u3092\u52b9\u679c\u7684\u306b\u4f4e\u4e0b\u3055\u305b\u3001\u30de\u30a6\u30b9\u304a\u3088\u3073\u975e\u30d2\u30c8\u970a\u9577\u985e\u30e2\u30c7\u30eb\u306b\u304a\u3044\u3066\u75c5\u72b6\u3092\u8efd\u6e1b\u3057\u3001\u5bff\u547d\u3092\u5ef6\u3070\u3059\u3053\u3068\u304c\u793a\u3055\u308c\u307e\u3057\u305f\u3002\u3057\u304b\u3082\u3001\u526f\u4f5c\u7528\u306f\u8a8d\u3081\u3089\u308c\u3066\u3044\u307e\u305b\u3093\u3002\u3053\u306e\u7d50\u679c\u306f\u3001\u9032\u884c\u4e2d\u306e\u81e8\u5e8a\u8a66\u9a13\u3068HG204\u304cMDS\u306e\u6cbb\u7642\u9078\u629e\u80a2\u3068\u3057\u3066\u6709\u671b\u3067\u3042\u308b\u3053\u3068\u3078\u306e\u4fe1\u983c\u3092\u5f37\u5316\u3059\u308b\u3082\u306e\u3067\u3059\u3002<\/p>\n\n\n\n<p><a href=\"https:\/\/www.nature.com\/articles\/s41593-024-01838-6\" target=\"_blank\" rel=\"noreferrer noopener\">\u51fa\u7248\u7269\u3078<\/a><\/p>\n\n\n\n<p>HERO\u81e8\u5e8a\u8a66\u9a13\u306f\u3001MECP2\u91cd\u8907\u75c7\u5019\u7fa4\u306e\u6cbb\u7642\u6cd5\u306e\u63a2\u6c42\u306b\u304a\u3051\u308b\u91cd\u8981\u306a\u4e00\u6b69\u3067\u3059\u3002\u6700\u5148\u7aef\u306eCRISPR Cas13\u6280\u8853\u3068\u6709\u671b\u306a\u4e88\u5099\u7684\u7d50\u679c\u306e\u7d44\u307f\u5408\u308f\u305b\u306f\u3001\u3053\u306e\u75be\u60a3\u306b\u82e6\u3057\u3080\u60a3\u8005\u3055\u3093\u3084\u3054\u5bb6\u65cf\u306b\u5e0c\u671b\u3092\u3082\u305f\u3089\u3057\u3066\u3044\u307e\u3059\u3002\u4eca\u5f8c\u6570\u30f6\u6708\u9593\u306f\u3001HG204 \u306e\u5b89\u5168\u6027\u3068\u6709\u52b9\u6027\u3092\u5341\u5206\u306b\u78ba\u8a8d\u3059\u308b\u305f\u3081\u306b\u3001\u7d99\u7d9a\u7684\u306a\u76e3\u8996\u3068\u7814\u7a76\u304c\u975e\u5e38\u306b\u91cd\u8981\u3068\u306a\u308a\u307e\u3059\u3002&nbsp;<\/p>","protected":false},"excerpt":{"rendered":"<p>HERO \u81e8\u5e8a\u8a66\u9a13\u306f\u3001MECP2 \u91cd\u8907\u75c7\u5019\u7fa4\uff08MDS\uff09\u3092\u5bfe\u8c61\u306b\u3001\u65b0\u3057\u3044 CRISPR Cas13 RNA \u7de8\u96c6\u7642\u6cd5\u3067\u3042\u308b HG204 \u3092\u8a55\u4fa1\u3059\u308b\u5148\u99c6\u7684\u306a\u7814\u7a76\u3067\u3059\u3002HuidaGene \u793e\u304c\u30b9\u30dd\u30f3\u30b5\u30fc\u3092\u52d9\u3081\u308b\u3053\u306e\u8a66\u9a13\u306f\u3001 MDS \u6cbb\u7642\u6cd5\u306e\u63a2\u6c42\u306b\u304a\u3044\u3066\u91cd\u8981\u306a\u4e00\u6b69\u3068\u306a\u308a\u307e\u3059\u30022024\u5e7411\u6708\u306b\u306f\u3001\u6700\u521d\u306e\u60a3\u8005\u306b HG204 \u306e\u6295\u4e0e\u306b\u6210\u529f\u3057\u3001MDS \u30b3\u30df\u30e5\u30cb\u30c6\u30a3\u306b\u65b0\u305f\u306a\u5e0c\u671b\u3092\u3082\u305f\u3089\u3057\u307e\u3057\u305f\u3002 <\/p>","protected":false},"author":73,"featured_media":1571,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_jf_save_progress":"","footnotes":""},"categories":[12,9],"tags":[],"class_list":["post-1562","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-crispr","category-research"],"_links":{"self":[{"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/posts\/1562","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/users\/73"}],"replies":[{"embeddable":true,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/comments?post=1562"}],"version-history":[{"count":1,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/posts\/1562\/revisions"}],"predecessor-version":[{"id":1563,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/posts\/1562\/revisions\/1563"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/media\/1571"}],"wp:attachment":[{"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/media?parent=1562"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/categories?post=1562"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/tags?post=1562"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}