{"id":1404,"date":"2023-12-16T17:30:00","date_gmt":"2023-12-16T16:30:00","guid":{"rendered":"https:\/\/remeds.org\/?p=1404"},"modified":"2025-02-06T18:04:25","modified_gmt":"2025-02-06T17:04:25","slug":"huidagene-a-promising-crispr-cas13-technology-from-shanghai-china","status":"publish","type":"post","link":"https:\/\/remeds.org\/ja\/2023\/12\/16\/huidagene-a-promising-crispr-cas13-technology-from-shanghai-china\/","title":{"rendered":"Huidagene\uff1a\u4e0a\u6d77\uff08\u4e2d\u56fd\uff09\u306e\u6709\u671b\u306aCRISPR-CAS13\u6280\u8853"},"content":{"rendered":"<p>Huidagene Therapeutics\u793e\u306f\u3001\u4e0a\u6d77\u3068\u30cb\u30e5\u30fc\u30b8\u30e3\u30fc\u30b8\u30fc\u306b\u62e0\u70b9\u3092\u7f6e\u304f\u30d0\u30a4\u30aa\u30c6\u30af\u30ce\u30ed\u30b8\u30fc\u4f01\u696d\u3067\u3001CRISPR\u6280\u8853\u3092\u57fa\u76e4\u3068\u3057\u305f\u65b0\u898f\u533b\u85ac\u54c1\u306e\u767a\u898b\u3001\u8a2d\u8a08\u3001\u958b\u767a\u306b\u53d6\u308a\u7d44\u3093\u3067\u3044\u307e\u3059\u3002\u540c\u793e\u306f\u3001\u7b4b\u75be\u60a3\u3001\u773c\u75be\u60a3\u3001\u795e\u7d4c\u75be\u60a3\u306b\u6ce8\u529b\u3057\u3066\u3044\u307e\u3059\u3002<\/p>\n\n\n\n<p>Huidagene\u793e\u306f\u3001\u65b0\u898f\u306eCRISPR-Cas13\u8907\u5408\u4f53\u3092\u7d30\u80de\u306b\u5c0e\u5165\u3059\u308b\u30a2\u30c7\u30ce\u968f\u4f34\u30a6\u30a4\u30eb\u30b9\uff08AAV\uff09\u30d9\u30af\u30bf\u30fcHG204\u3092\u958b\u767a\u3057\u305f\u3002MECP2\u91cd\u8907\u75c7\u5019\u7fa4\u306e\u305f\u3081\u306b\u30c7\u30b6\u30a4\u30f3\u3055\u308c\u305f\u3001&nbsp;<strong>HG204\u306f\u3001\u904e\u5270\u767a\u73fe\u3057\u305fMECP2 RNA\u306e\u5206\u89e3\u3092\u8a98\u5c0e\u3059\u308b\u3053\u3068\u3067\u3001MeCP2\u30bf\u30f3\u30d1\u30af\u8cea\u306e\u904e\u5270\u7523\u751f\u3092\u5236\u5fa1\u3059\u308b\u3053\u3068\u3092\u76ee\u7684\u3068\u3057\u3066\u3044\u308b\u3002<\/strong>&nbsp;\u3053\u306e\u9769\u65b0\u7684\u306a\u65b0\u85ac\u5019\u88dc\u306f\u30011\u56de\u306e\u6295\u4e0e\u3067MDS\u3092\u6cbb\u7652\u3055\u305b\u308b\u53ef\u80fd\u6027\u3092\u79d8\u3081\u3066\u3044\u308b\u3002<\/p>\n\n\n\n<p>\u79c1\u305f\u3061\u306e\u7d44\u7e54\u306f\u3001\u3053\u306e\u9769\u65b0\u7684\u306a\u30c6\u30af\u30ce\u30ed\u30b8\u30fc\u3068\u305d\u308c\u304c\u5c06\u6765\u7684\u306b\u4f55\u3092\u610f\u5473\u3059\u308b\u306e\u304b\u3092\u77e5\u308b\u305f\u3081\u306b\u3001Huidagene\u793e\u306e\u30ea\u30fc\u30c0\u30fc\u305f\u3061\u3068\u5b9f\u308a\u3042\u308b\u8a71\u3057\u5408\u3044\u304c\u51fa\u6765\u307e\u3057\u305f\u3002&nbsp;<strong>\u30d2\u30c8\u5316MDS\u30de\u30a6\u30b9\u30e2\u30c7\u30eb\u3067HG204\u3092\u7528\u3044\u305f\u524d\u81e8\u5e8a\u8a66\u9a13\u3067\u306f\u3001MeCP2\u30bf\u30f3\u30d1\u30af\u8cea\u306e\u6709\u610f\u306a\u6e1b\u5c11\u3068\u75c7\u72b6\u306e\u6539\u5584\u304c\u793a\u3055\u308c\u307e\u3057\u305f\u3002<\/strong>&nbsp;\u3053\u308c\u3089\u306e\u7d50\u679c\u306f\u5e0c\u671b\u3092\u3082\u305f\u3089\u3059\u3082\u306e\u3067\u3059\u304c\u3001MECP2\u30bf\u30f3\u30d1\u30af\u8cea\u30ec\u30d9\u30eb\u306e\u8abf\u7bc0\u5f8c\u306bMDS\u306e\u75c7\u72b6\u304c\u53ef\u9006\u7684\u3067\u3042\u308b\u3053\u3068\u3092\u793a\u30592\u4f8b\u76ee\u306e\u8a3c\u660e\u3067\u3082\u3042\u308a\u307e\u3059\uff08\u30d5\u30fc\u30c0\u30fb\u30be\u30fc\u30d3\u6559\u6388\u3068\u30c1\u30fc\u30e0\u306b\u3088\u308bASO\u306e\u6295\u4e0e\u306b\u3088\u3063\u3066\u4ee5\u524d\u306b\u5b9f\u8a3c\u3055\u308c\u3066\u3044\u307e\u3057\u305f\uff09\u3002<\/p>\n\n\n\n<p><strong>HuidaGene\u793e\u306f\u3001HG204\u306b\u95a2\u3059\u308b\u524d\u81e8\u5e8a\u7814\u7a76\u306e\u6700\u7d42\u6bb5\u968e\u306b\u5165\u3063\u3066\u3044\u3066\u30012024\u5e74\u534a\u3070\u306b\u958b\u59cb\u4e88\u5b9a\u306e\u4e2d\u56fd\u306e\u60a3\u8005\u3092\u5bfe\u8c61\u3068\u3057\u305f\u81e8\u5e8a\u8a66\u9a13\u306e\u6e96\u5099\u3092\u9032\u3081\u3066\u3044\u307e\u3059\u3002&nbsp;<\/strong>\u3053\u306e\u81e8\u5e8a\u8a66\u9a13\u304c\u6210\u529f\u3059\u308c\u3070\u30012025\u5e74\u304b\u30892026\u5e74\u306b\u304b\u3051\u3066\u3001\u30a2\u30e1\u30ea\u30ab\u3084\u30e8\u30fc\u30ed\u30c3\u30d1\u306a\u3069\u4ed6\u306e\u56fd\u3067\u306e\u81e8\u5e8a\u8a66\u9a13\u3092\u8a08\u753b\u3057\u3066\u3044\u307e\u3059\u3002<\/p>\n\n\n\n<p><br>10\u670831\u65e5\u3001FDA\uff08\u7c73\u56fd\u98df\u54c1\u533b\u85ac\u54c1\u5c40\uff09\u306fHG204\u306b\u3001\u300c\u5e0c\u5c11\u5c0f\u5150\u75be\u60a3\u300d\u3068\u300c\u5e0c\u5c11\u75be\u75c5\u7528\u533b\u85ac\u54c1\u300d\u306e\u4e21\u65b9\u306e\u6307\u5b9a\u3092\u4e0e\u3048\u307e\u3057\u305f\u3002\u3053\u306e\u85ac\u4e8b\u6307\u5b9a\u306f\u3001\u4e2d\u56fd\u56fd\u5916\u3067\u306e\u81e8\u5e8a\u8a66\u9a13\u306b\u5411\u3051\u305f\u7b2c\u4e00\u6b69\u3067\u3042\u308a\u3001\u533b\u85ac\u54c1\u767b\u9332\u6642\u306e\u66f8\u985e\u5be9\u67fb\u306e\u8fc5\u901f\u5316\u3092\u53ef\u80fd\u306b\u3059\u308b\u3082\u306e\u3067\u3082\u3042\u308a\u307e\u3059\u3002\u3053\u306e\u6717\u5831\u306f\u3001\u5168\u304f\u4e88\u671f\u3057\u3066\u3044\u306a\u3044\u3082\u306e\u3067\u3057\u305f\u304c\u3001\u5730\u57df\u793e\u4f1a\u306b\u3055\u3089\u306a\u308b\u5e0c\u671b\u3092\u3082\u305f\u3089\u3059\u3082\u306e\u3068\u306a\u308a\u307e\u3057\u305f\u3002<\/p>\n\n\n\n<p>\u79c1\u305f\u3061\u306fHuidaGene\u793e\u3068\u9023\u7d61\u3092\u53d6\u308a\u7d9a\u3051\u3001\u65b0\u305f\u306a\u9032\u5c55\u304c\u3042\u308c\u3070\u30b3\u30df\u30e5\u30cb\u30c6\u30a3\u306e\u7686\u69d8\u306b\u60c5\u5831\u3092\u63d0\u4f9b\u3057\u3066\u3044\u304d\u307e\u3059\u3002<\/p>\n\n\n\n<figure class=\"wp-block-image\"><img decoding=\"async\" src=\"https:\/\/dupmecp2.eu\/wp-content\/uploads\/2023\/12\/Huidagene_Pipeline_2023_white-edited.png\" alt=\"\" class=\"wp-image-22144\"\/><\/figure>\n\n\n\n<p><a href=\"https:\/\/www.huidagene.com\/new\/news\/48\" target=\"_blank\" rel=\"noreferrer noopener\">\u30d7\u30ec\u30b9\u30ea\u30ea\u30fc\u30b9\u3078<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Huidagene Therapeutics is a biotechnology company based in Shanghai and New Jersey, focusing on discovering, engineering, and developing novel CRISPR-based medicines. The company focuses on muscular, ophthalmic and neurological disorders. Huidagene has developed HG204, an adeno-associated viral (AAV) vector delivering a novel CRISPR-Cas13 complex into cells. Designed for MECP2 duplication syndrome,&nbsp;HG204 aims to regulate the overproduction of the MeCP2 protein levels by inducing the degradation of overexpressed MECP2 RNA.&nbsp;This innovative drug candidate holds the potential to cure MDS through a single administration. Our organization has had productive discussions with Huidagene\u2019s leaders to learn more about this innovative technology and what it means for the future.&nbsp;Preclinical studies using HG204 in a [&hellip;]<\/p>\n","protected":false},"author":73,"featured_media":1429,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_jf_save_progress":"","footnotes":""},"categories":[12,10,9],"tags":[],"class_list":["post-1404","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-crispr","category-cure","category-research"],"_links":{"self":[{"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/posts\/1404","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=1404"}],"version-history":[{"count":1,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/posts\/1404\/revisions"}],"predecessor-version":[{"id":1405,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/posts\/1404\/revisions\/1405"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/media\/1429"}],"wp:attachment":[{"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/media?parent=1404"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/categories?post=1404"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/tags?post=1404"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}