{"id":4576,"date":"2025-09-25T16:23:32","date_gmt":"2025-09-25T13:23:32","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4576"},"modified":"2025-09-25T16:23:33","modified_gmt":"2025-09-25T13:23:33","slug":"spot-mrna03-gene-therapy-early-phase-1-clinical-trial-nct07188012","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/spot-mrna03-gene-therapy-early-phase-1-clinical-trial-nct07188012\/","title":{"rendered":"Duchenne Kas Distrofisi i\u00e7in SPOT-mRNA03 Gen Terapisi Erken Faz 1 Klinik \u00c7al\u0131\u015fmas\u0131na Ba\u015fl\u0131yor"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Shanghai Siponuoyin Biyoteknoloji taraf\u0131ndan Duchenne Kas Distrofisi (DMD) i\u00e7in geli\u015ftirilen SPOT-mRNA03 gen terapisinin erken faz 1 \u00e7al\u0131\u015fmas\u0131 (NCT07188012), distrofin'in iskelet kaslar\u0131nda uygulanmas\u0131ndan sonraki g\u00fcvenli\u011fini ve ekspresyonunu inceleyen bir pilot \u00e7al\u0131\u015fmad\u0131r.<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u0130\u00e7indekiler<\/h2><nav><ul><li><a href=\"#what-is-spot-m-rna-03-gene-therapy\">SPOT-mRNA03 Gen Terapisi Nedir?<\/a><\/li><li><a href=\"#spot-m-rna-03-gene-therapy-early-phase-1-nct-07188012\">SPOT-mRNA03 Gen Terapisi Erken Faz 1 \u2013 NCT07188012<\/a><\/li><li><a href=\"#how-to-participate-in-the-sgt-003-clinical-trial\">SPOT-mRNA03 Klinik \u00c7al\u0131\u015fmas\u0131na Nas\u0131l Kat\u0131l\u0131rs\u0131n\u0131z?<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-spot-m-rna-03-gene-therapy\"><strong>SPOT-mRNA03 Gen Terapisi Nedir?<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>SPOT-mRNA03, Duchenne Musk\u00fcler Distrofi (DMD) i\u00e7in gen tedavisi \u00fcr\u00fcn\u00fcd\u00fcr.<\/strong> DMD hastalar\u0131na uyguland\u0131ktan sonra, EV&#039;lerin membran\u0131ndaki molek\u00fcler hedefleme peptidi, SPOT-mRNA03&#039;yi hastan\u0131n kas h\u00fccrelerine y\u00f6nlendirebilir ve b\u00f6ylece h\u00fccre i\u00e7i ribozomlar ve amino asitler kullan\u0131larak distrofin eksikli\u011fi olan resept\u00f6r h\u00fccreleri taraf\u0131ndan endojen tam uzunluktaki intakt distrofinin translasyonunu ve salg\u0131lanmas\u0131n\u0131 sa\u011flayabilir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"spot-m-rna-03-gene-therapy-early-phase-1-nct-07188012\"><strong>SPOT-mRNA03 Gen Terapisi Erken Faz 1 \u2013 NCT07188012<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Bu, DMD hastalar\u0131na intraven\u00f6z olarak verilen SPOT-mRNA03&#039;yi kullanan tek kollu, a\u00e7\u0131k etiketli, FIH, tek merkezli bir ke\u015fifsel klinik ara\u015ft\u0131rmad\u0131r. Tam uzunlukta distrofin mRNA&#039;s\u0131, SPOT-mRNA03 ad\u0131 verilen kas hedefli h\u00fccre d\u0131\u015f\u0131 vezik\u00fcllerde (EV&#039;ler) bulunur. EV zar\u0131ndaki molek\u00fcler hedefleme peptitleri, SPOT-mRNA03&#039;ye hedefleme kabiliyeti kazand\u0131r\u0131r ve bu da distrofin mRNA&#039;s\u0131n\u0131n DMD i\u00e7in bir gen tedavisi \u00fcr\u00fcn\u00fc olarak da\u011f\u0131t\u0131lmas\u0131n\u0131 m\u00fcmk\u00fcn k\u0131lar.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fma Ba\u015flang\u0131c\u0131 (Tahmini)<\/strong>: 2025-08-06<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Birincil Tamamlanma (Tahmini)<\/strong>: 2026-12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fman\u0131n Tamamlanmas\u0131 (Tahmini)<\/strong>: 2026-12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Kat\u0131l\u0131mc\u0131 Say\u0131s\u0131 (Tahmini)<\/strong>: 6<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fma T\u00fcr\u00fc<\/strong>: Erken Faz 1<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Konum<\/strong>: <a href=\"https:\/\/maps.app.goo.gl\/Z2CyEqLydJ4Kxh9aA\" target=\"_blank\" rel=\"noreferrer noopener\">\u015eanghay \u00c7ocuk T\u0131p Merkezi<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fmaya Uygun Ya\u015flar<\/strong>: 2 Ya\u015f\u0131ndan 6 Ya\u015f\u0131na Kadar (Y\u00fcr\u00fcyebilen Erkekler)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Temel dahil etme ve hari\u00e7 tutma kriterleri. \u2013 <a href=\"https:\/\/clinicaltrials.gov\/study\/NCT07188012\" target=\"_blank\" rel=\"noreferrer noopener\">NCT07188012<\/a> \u2013<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"how-to-participate-in-the-sgt-003-clinical-trial\"><strong>SPOT-mRNA03 Klinik \u00c7al\u0131\u015fmas\u0131na Nas\u0131l Kat\u0131l\u0131rs\u0131n\u0131z?<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">SPOT-mRNA03 gen tedavisi erken faz 1 klinik \u00e7al\u0131\u015fmas\u0131na kat\u0131lmak i\u00e7in a\u015fa\u011f\u0131daki ileti\u015fim bilgilerini kullanabilirsiniz.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0130sim<\/strong>: Winston Kasabas\u0131<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Telefon Numaras\u0131<\/strong>: 212-920-5501<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>E-posta<\/strong>: Winston.town@spotbiosystems.com<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>The early phase 1 study (NCT07188012) of SPOT-mRNA03 gene therapy for Duchenne Muscular Dystrophy (DMD) developed by Shanghai Siponuoyin Biotechnology is a pilot study of the safety and expression of dystrophin in skeletal muscles after administration. What is SPOT-mRNA03 Gene Therapy SPOT-mRNA03 is the gene therapy product for Duchenne Muscular Dystrophy (DMD). Following administration to [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4580,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[123,137,242,359,61,424,423,425],"class_list":["post-4576","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-china","tag-clinical-trial","tag-clinical-trials","tag-full-length-dystrophin","tag-gene-therapy","tag-shanghai-siponuoyin","tag-spot-mrna03","tag-spot-mrna03-gene-therapy"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4576","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=4576"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4576\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4580"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4576"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4576"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4576"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}