{"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\/tr\/2023\/12\/16\/huidagene-a-promising-crispr-cas13-technology-from-shanghai-china\/","title":{"rendered":"Huidagene: \u015eangay'dan (\u00c7in) umut verici bir CRISPR-CAS13 teknolojisi"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Huidagene Therapeutics, \u015eanghay ve New Jersey merkezli, yeni CRISPR tabanl\u0131 ila\u00e7lar ke\u015ffetmeye, tasarlamaya ve geli\u015ftirmeye odaklanan bir biyoteknoloji \u015firketidir. \u015eirket, kas, g\u00f6z ve n\u00f6rolojik hastal\u0131klara odaklanmaktad\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Huidagene, h\u00fccrelere yeni bir CRISPR-Cas13 kompleksini ta\u015f\u0131yan bir adeno-ili\u015fkili viral (AAV) vekt\u00f6r olan HG204\u2019\u00fc geli\u015ftirdi. MECP2 duplikasyon sendromu i\u00e7in tasarlanan&nbsp;<strong>HG204, a\u015f\u0131r\u0131 eksprese edilen MECP2 RNA\u2019s\u0131n\u0131n y\u0131k\u0131m\u0131n\u0131 ind\u00fckleyerek MeCP2 protein seviyelerindeki a\u015f\u0131r\u0131 \u00fcretimi d\u00fczenlemeyi ama\u00e7lamaktad\u0131r.<\/strong>&nbsp;Bu yenilik\u00e7i ila\u00e7 aday\u0131, tek bir uygulama yoluyla MDS'yi tedavi etme potansiyeline sahiptir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Kurulu\u015fumuz, bu yenilik\u00e7i teknoloji ve bunun gelecek i\u00e7in ne anlama geldi\u011fi hakk\u0131nda daha fazla bilgi edinmek i\u00e7in Huidagene'in liderleriyle verimli g\u00f6r\u00fc\u015fmeler ger\u00e7ekle\u015ftirdi.&nbsp;<strong>\u0130nsanla\u015ft\u0131r\u0131lm\u0131\u015f bir MDS fare modelinde HG204 kullan\u0131larak yap\u0131lan klinik \u00f6ncesi \u00e7al\u0131\u015fmalar, MeCP2 proteininde \u00f6nemli bir azalma ve semptomlar\u0131n tersine d\u00f6nd\u00fc\u011f\u00fcn\u00fc g\u00f6stermi\u015ftir.<\/strong>&nbsp;Bunlar cesaret verici sonu\u00e7lar olmakla birlikte, bu ayn\u0131 zamanda MECP2 protein seviyelerinin d\u00fczenlenmesinden sonra MDS semptomlar\u0131n\u0131n tersine \u00e7evrilebilirli\u011finin ikinci kan\u0131t\u0131d\u0131r (daha \u00f6nce Prof. H. Zoghbi &amp; Ekibi taraf\u0131ndan ASO'nun uygulanmas\u0131yla g\u00f6sterildi\u011fi gibi).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Huidagene, HG204 ile klinik \u00f6ncesi \u00e7al\u0131\u015fmalar\u0131 tamamlamak ve 2024 ortas\u0131nda \u00c7in\u2019de hastalar i\u00e7in ba\u015flayacak bir klinik \u00e7al\u0131\u015fmaya haz\u0131rlanma s\u00fcrecindedir.&nbsp;<\/strong>Huidagene er i gang med at afslutte pr\u00e6kliniske studier med HG204 og forbereder sig p\u00e5 et klinisk fors\u00f8g med patienter i Kina, der er planlagt til at begynde i midten af 2024.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><br>31 Ekim\u2019de, ABD\u2019nin sa\u011fl\u0131k otoritesi olan FDA (G\u0131da ve \u0130la\u00e7 Dairesi), HG204\u2019e hem Nadir Pediatrik Hastal\u0131k hem de Yetim \u0130la\u00e7 Tan\u0131m\u0131 vermi\u015ftir.\nBu d\u00fczenleyici tan\u0131m, \u00c7in d\u0131\u015f\u0131nda yap\u0131lacak bir klinik \u00e7al\u0131\u015fmaya y\u00f6nelik ilk ad\u0131md\u0131r ve ilac\u0131n kayd\u0131 s\u0131ras\u0131nda dosyan\u0131n h\u0131zland\u0131r\u0131lm\u0131\u015f incelemesini m\u00fcmk\u00fcn k\u0131lacakt\u0131r.\nBu haber tamamen beklenmedik olsa da, topluluk i\u00e7in ek bir umut getirmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ekibimiz Huidagene ile ileti\u015fimde kalacak ve toplulu\u011fumuzu yeni geli\u015fmeler hakk\u0131nda bilgilendirmeye devam edecektir.<\/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 class=\"wp-block-paragraph\"><a href=\"https:\/\/www.huidagene.com\/new\/news\/48\" target=\"_blank\" rel=\"noreferrer noopener\">Bas\u0131n b\u00fcltenine<\/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 [&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\/tr\/wp-json\/wp\/v2\/posts\/1404","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/users\/73"}],"replies":[{"embeddable":true,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/comments?post=1404"}],"version-history":[{"count":1,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/posts\/1404\/revisions"}],"predecessor-version":[{"id":1405,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/posts\/1404\/revisions\/1405"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/media\/1429"}],"wp:attachment":[{"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/media?parent=1404"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/categories?post=1404"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/tags?post=1404"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}