{"id":3802,"date":"2025-05-05T17:54:31","date_gmt":"2025-05-05T14:54:31","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3802"},"modified":"2025-05-05T17:55:03","modified_gmt":"2025-05-05T14:55:03","slug":"scribe-therapeutics-and-prevail-therapeutics-to-share-in-vivo-crispr-based-genome-editing-data-at-asgct-2025","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/scribe-therapeutics-and-prevail-therapeutics-to-share-in-vivo-crispr-based-genome-editing-data-at-asgct-2025\/","title":{"rendered":"Scribe Therapeutics ve Prevail Therapeutics, ASGCT 2025&#039;te Canl\u0131da CRISPR Tabanl\u0131 Genom D\u00fczenleme Verilerini Payla\u015facak"},"content":{"rendered":"<p class=\"wp-block-paragraph\">CRISPR&#039;\u0131n insan sa\u011fl\u0131\u011f\u0131n\u0131 d\u00f6n\u00fc\u015ft\u00fcrme potansiyelini ortaya \u00e7\u0131karan bir genetik ila\u00e7 \u015firketi olan Scribe Therapeutics, bug\u00fcn 13-17 May\u0131s 2025 tarihleri aras\u0131nda New Orleans, LA&#039;daki Ernest N. Morial Kongre Merkezi&#039;nde ger\u00e7ekle\u015fecek olan 28. Amerikan Gen ve H\u00fccre Terapisi Derne\u011fi (ASGCT) Y\u0131ll\u0131k Toplant\u0131s\u0131&#039;na kat\u0131laca\u011f\u0131n\u0131 duyurdu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Scribe Therapeutics, \u015firketin in vivo yeni genetik ila\u00e7lar geli\u015ftirmek i\u00e7in CRISPR yakla\u015f\u0131m\u0131yla sa\u011flad\u0131\u011f\u0131 devam eden geli\u015fmeleri sergileyen iki sunum yapacak.<\/strong> G\u00fc\u00e7l\u00fc terap\u00f6tik uygulaman\u0131n bir g\u00f6steriminde, Scribe Therapeutics, solunum ve kalp yetmezli\u011fine yol a\u00e7abilen ciddi bir n\u00f6rom\u00fcsk\u00fcler bozukluk olan Duchenne Musk\u00fcler Distrofisi&#039;ndeki (DMD) mutasyonlar\u0131 hedeflemek i\u00e7in X-Edit\u00f6r (XE) teknolojisini kullanan yeni genom d\u00fczenleme stratejileri sunacakt\u0131r. Bu \u00e7al\u0131\u015fmadan elde edilen bulgular, XE&#039;yi DMD ve di\u011fer kal\u0131tsal kas bozukluklar\u0131n\u0131n tedavisi i\u00e7in etkili ve \u00e7ok y\u00f6nl\u00fc bir platform olarak sergileyecektir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ortak sunumda, Eli Lilly and Company&#039;nin tamamen sahip oldu\u011fu bir yan kurulu\u015f olan Scribe Therapeutics ve Prevail Therapeutics&#039;nin, Scribe Therapeutics&#039;nin CRISPR tabanl\u0131 XE&#039;sini i\u00e7eren kendi kendini inaktive eden adeno ili\u015fkili viral (AAV) vekt\u00f6rlerini tasarlamak i\u00e7in yapt\u0131\u011f\u0131 \u00e7al\u0131\u015fmalar ve farelerin merkezi sinir sisteminde g\u00fc\u00e7l\u00fc, ge\u00e7ici ve kontrol edilebilir hedefli in vivo d\u00fczenlemeyi g\u00f6steren sonu\u00e7lar a\u00e7\u0131klanacakt\u0131r. \u015eirketler yak\u0131n zamanda n\u00f6rolojik ve n\u00f6rom\u00fcsk\u00fcler hastal\u0131klar\u0131 ele almak i\u00e7in yapt\u0131klar\u0131 i\u015f birli\u011finde bir ba\u015far\u0131 kilometre ta\u015f\u0131na ula\u015ft\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Devam\u0131n\u0131 oku: <a href=\"https:\/\/dmdwarrior.com\/tr\/new-gene-therapies-for-duchenne-muscular-dystrophy\/\">Duchenne Musk\u00fcler Distrofisi \u0130\u00e7in Yeni Gen Terapileri<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Scribe Therapeutics, a genetic medicines company unlocking the potential of CRISPR to transform human health, today announced its participation in the 28th American Society of Gene &amp; Cell Therapy (ASGCT) Annual Meeting taking place May 13-17, 2025, at the Ernest N. Morial Convention Center in New Orleans, LA. Scribe Therapeutics will deliver two presentations that [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3803,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[366,290,72,61,365,364,363],"class_list":["post-3802","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-asgct-2025","tag-crispr","tag-gene-editing","tag-gene-therapy","tag-genome-editing","tag-prevail-therapeutics","tag-scribe-therapeutics"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3802","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=3802"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3802\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3803"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3802"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3802"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3802"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}