{"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\/tr\/2025\/03\/12\/recruitment-starts-for-hero-crispr-cas13-clinical-trial-for-mds\/","title":{"rendered":"HERO i\u00e7in Kat\u0131l\u0131mc\u0131 Al\u0131m\u0131 Ba\u015flad\u0131: MDS\u2019ye Y\u00f6nelik CRISPR-Cas13 Klinik \u00c7al\u0131\u015fmas\u0131"},"content":{"rendered":"<p><a href=\"https:\/\/dupmecp2.eu\/crispr-cas13\/\" target=\"_blank\" rel=\"noreferrer noopener\">Teknoloji hakk\u0131nda<\/a><\/p>\n\n\n\n<p><strong>Denemeye Genel Bak\u0131\u015f<\/strong>&nbsp;<\/p>\n\n\n\n<p>HERO denemesi, tedavinin do\u011frudan beyne uygulanmas\u0131n\u0131 sa\u011flayan intrasebroventrik\u00fcler yolla tek seferlik HG204 enjeksiyonunu i\u00e7ermektedir. \u00c7al\u0131\u015fma i\u00e7in hasta al\u0131m\u0131 Ekim 2024\u2019te ba\u015flam\u0131\u015f olup, MECP2 Duplikasyon Sendromu tan\u0131s\u0131 do\u011frulanm\u0131\u015f 2 ila 18 ya\u015f aras\u0131 alt\u0131 erkek kat\u0131l\u0131mc\u0131n\u0131n dahil edilmesi hedeflenmektedir. Her kat\u0131l\u0131mc\u0131, 8 haftal\u0131k tarama d\u00f6nemi, tedavi uygulamas\u0131 ve 52 haftal\u0131k takip a\u015famas\u0131ndan olu\u015fan 60 haftal\u0131k \u00e7al\u0131\u015fmaya kat\u0131lacakt\u0131r. Birincil ama\u00e7lar, advers olaylar\u0131 izlemek ve tedavi sonras\u0131 geli\u015fimsel ilerlemeyi de\u011ferlendirmektir. \u00c7al\u0131\u015fma h\u00e2lihaz\u0131rda \u00c7in\u2019in Pekin kentindeki Peking University Hospital\u2019da y\u00fcr\u00fct\u00fclmektedir. \u00c7in\u2019de daha fazla hasta al\u0131m\u0131, \u00e7al\u0131\u015fmada tedavi edilen ilk hastalardan elde edilen sonu\u00e7lara ba\u011fl\u0131 olacakt\u0131r.&nbsp;<\/p>\n\n\n\n<p>Daha ayr\u0131nt\u0131l\u0131 bilgi i\u00e7in sizi \u00e7al\u0131\u015fma protokol\u00fcn\u00fc okumaya davet ediyoruz.<\/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\">\u00c7al\u0131\u015fma protokol\u00fcne buradan eri\u015febilirsiniz<\/a><\/p>\n\n\n\n<p><strong>\u0130lk Doz Uygulamas\u0131 &amp; \u00d6n Bulgular<\/strong>&nbsp;<\/p>\n\n\n\n<p>Kas\u0131m ay\u0131nda HuidaGene, ilk hastaya HG204\u2019\u00fcn ba\u015far\u0131yla uyguland\u0131\u011f\u0131n\u0131 duyurdu. Enjeksiyondan alt\u0131 hafta sonra hastada \u00f6nemli bir yan etki g\u00f6r\u00fclmedi ve etkinli\u011fe dair \u00f6n bulgular g\u00f6zlemlendi. \u00d6n\u00fcm\u00fczdeki haftalarda, tedavinin g\u00fcvenli\u011fi ve etkinli\u011fini daha ayr\u0131nt\u0131l\u0131 de\u011ferlendirmek i\u00e7in hasta yak\u0131ndan izlenecektir.&nbsp;<\/p>\n\n\n\n<p><strong>Sonraki ad\u0131mlar ve hedefler<\/strong>&nbsp;<\/p>\n\n\n\n<p>HuidaGene, d\u00fczenleyici onaylarda \u00f6nemli ilerleme kaydetmi\u015f ve MECP2 Duplikasyon Sendromu tedavisinde HG204 i\u00e7in iki \u00f6nemli FDA stat\u00fcs\u00fc alm\u0131\u015ft\u0131r: Nadir Pediatrik Hastal\u0131k Stat\u00fcs\u00fc (RPDD) ve Yetim \u0130la\u00e7 Stat\u00fcs\u00fc (ODD). HERO \u00e7al\u0131\u015fmas\u0131 \u00c7in\u2019de y\u00fcr\u00fct\u00fclse de biyoteknoloji \u015firketi, ABD ve Avrupa\u2019da yap\u0131labilecek ileri \u00e7al\u0131\u015fmalar i\u00e7in FDA ve EMA ile de g\u00f6r\u00fc\u015fmelere ba\u015flam\u0131\u015ft\u0131r.&nbsp;<\/p>\n\n\n\n<p><strong>Yay\u0131nlanm\u0131\u015f Ara\u015ft\u0131rma<\/strong>&nbsp;<\/p>\n\n\n\n<p class=\"translation-block\">Bu arada HuidaGene, HG204 de dahil olmak \u00fczere CRISPR-Cas13 teknolojisine ili\u015fkin preklinik deneysel bulgular\u0131n ayr\u0131nt\u0131lar\u0131n\u0131 Nature Neuroscience dergisinde yay\u0131mlad\u0131. \u00c7al\u0131\u015fmalar, HG204\u2019\u00fcn MECP2 mRNA seviyelerini etkili bir \u015fekilde azaltt\u0131\u011f\u0131n\u0131, hastal\u0131k semptomlar\u0131n\u0131 hafifletti\u011fini ve yan etkilere yol a\u00e7madan fare ve insan d\u0131\u015f\u0131 primat modellerinde ya\u015fam s\u00fcresini uzatt\u0131\u011f\u0131n\u0131 g\u00f6stermi\u015ftir. Bu sonu\u00e7lar, devam eden klinik \u00e7al\u0131\u015fmaya ve HG204\u2019\u00fcn MDS i\u00e7in potansiyel bir tedavi se\u00e7ene\u011fi olmas\u0131na dair g\u00fcveni g\u00fc\u00e7lendirmektedir.<\/p>\n\n\n\n<p><a href=\"https:\/\/www.nature.com\/articles\/s41593-024-01838-6\" target=\"_blank\" rel=\"noreferrer noopener\">Yay\u0131na bak\u0131n\u0131z<\/a><\/p>\n\n\n\n<p>HERO klinik \u00e7al\u0131\u015fmas\u0131, MECP2 Duplikasyon Sendromu i\u00e7in bir tedavi aray\u0131\u015f\u0131nda \u00f6nemli bir ad\u0131m\u0131 temsil etmektedir. Son teknoloji CRISPR Cas13 teknolojisi ile umut verici \u00f6n bulgular\u0131n birle\u015fimi, bu sendromdan etkilenen hasta ve ailelere umut vermektedir. \u00d6n\u00fcm\u00fczdeki aylarda HG204\u2019\u00fcn MDS tedavisindeki g\u00fcvenli\u011fini ve etkinli\u011fini tam olarak belirlemek i\u00e7in s\u00fcrekli izleme ve ara\u015ft\u0131rmalar kritik \u00f6neme sahip olacakt\u0131r.&nbsp;<\/p>","protected":false},"excerpt":{"rendered":"<p>HERO klinik \u00e7al\u0131\u015fmas\u0131, MECP2 Duplikasyon Sendromu (MDS) i\u00e7in geli\u015ftirilen yeni bir CRISPR Cas13 RNA d\u00fczenleme tedavisi olan HG204\u2019\u00fc de\u011ferlendiren \u00f6nc\u00fc bir ara\u015ft\u0131rmad\u0131r. HuidaGene taraf\u0131ndan desteklenen bu \u00e7al\u0131\u015fma, MDS i\u00e7in bir tedavi aray\u0131\u015f\u0131nda \u00f6nemli bir ad\u0131m\u0131 temsil etmektedir. Kas\u0131m 2024\u2019te ilk hastaya ba\u015far\u0131yla HG204 uygulanm\u0131\u015f ve bu durum MDS toplulu\u011funa yeniden umut vermi\u015ftir. <\/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\/tr\/wp-json\/wp\/v2\/posts\/1562","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=1562"}],"version-history":[{"count":1,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/posts\/1562\/revisions"}],"predecessor-version":[{"id":1563,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/posts\/1562\/revisions\/1563"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/media\/1571"}],"wp:attachment":[{"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/media?parent=1562"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/categories?post=1562"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/tags?post=1562"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}