{"id":2723,"date":"2025-02-01T09:45:45","date_gmt":"2025-02-01T06:45:45","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=2723"},"modified":"2025-02-01T14:34:43","modified_gmt":"2025-02-01T11:34:43","slug":"a-novel-mini-crispr-could-be-more-successful-at-editing-the-dystrophin-gene","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/a-novel-mini-crispr-could-be-more-successful-at-editing-the-dystrophin-gene\/","title":{"rendered":"Yeni Bir \u201cMini-CRISPR\u201d Distrofin Genini D\u00fczenlemede Daha Ba\u015far\u0131l\u0131 Olabilir"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Birka\u00e7 ara\u015ft\u0131rma grubu, d\u00fczenleme yeteneklerini korurken tek bir AAV vekt\u00f6r\u00fcne s\u0131\u011fabilen kompakt CRISPR sistemleri yaratmaya \u00e7al\u0131\u015f\u0131yor. Mammoth Biosciences&#039;daki grup bunlardan biri; ba\u015far\u0131l\u0131 olmu\u015f olabilirler. Ara\u015ft\u0131rmac\u0131lar\u0131, mikroorganizmalarda bulunan \u00e7e\u015fitli CRISPR tiplerinden genetik dizi bilgilerini ay\u0131klayarak i\u015fe ba\u015flad\u0131lar.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirketin ba\u015f bilim sorumlusu ve kurucu orta\u011f\u0131 ve eski bir Doudna \u00f6\u011frencisi olan Lucas Harrington, ilk 176 ba\u015fvuruyu &quot;bir dizi testten&quot; ge\u00e7irdikten sonra &quot;samanl\u0131ktaki i\u011fneyi&quot; bulduklar\u0131n\u0131 iddia ediyor. En iyi se\u00e7ene\u011fe NanoCas ad\u0131 verildi; 425 amino asitle Cas9&#039;dan yakla\u015f\u0131k \u00fc\u00e7te bir daha uzun. Harrington ve ortaklar\u0131 taraf\u0131ndan memeli DNA&#039;s\u0131n\u0131 etkili bir \u015fekilde kesmek i\u00e7in tasarland\u0131.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">NanoCas ara\u015ft\u0131rmas\u0131n\u0131n temel \u00e7\u0131kar\u0131mlar\u0131 \u015funlard\u0131r:<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>D\u00fczenleme verimlili\u011fi birinci nesil CRISPR sistemleriyle e\u015fle\u015fiyor: Fare karaci\u011ferinde PCSK9 genini in vivo hedef al\u0131rken, NanoCas yakla\u015f\u0131k 60%&#039;lik doyurucu d\u00fczenleme verimlili\u011fi g\u00f6sterdi, bu da boyut olarak yakla\u015f\u0131k \u00fc\u00e7 kat daha b\u00fcy\u00fck olan SaCas9 ile ayn\u0131 seviyedeydi. Her iki CRISPR sistemi de serum PCSK9 proteinini tespit edilemeyen seviyelere d\u00fc\u015f\u00fcrd\u00fc.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Birden fazla kas dokusunda sa\u011flam tek AAV d\u00fczenlemesi: NanoCas, Duchenne Musk\u00fcler Distrofisi&#039;nin (DMD) insanla\u015ft\u0131r\u0131lm\u0131\u015f bir fare modelinde, tek bir AAV vekt\u00f6r\u00fc arac\u0131l\u0131\u011f\u0131yla verildi\u011finde, kuadriseps, bald\u0131r ve kalp kas\u0131 boyunca distrofin geninin 10% ila 40% d\u00fczenlemesini g\u00f6sterdi.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u0130nsan olmayan primatlarda tek AAV kas d\u00fczenlemesinin ilk g\u00f6sterimi: NanoCas, sinomolgus makaklar\u0131n\u0131n iskelet kas\u0131nda distrofin hedeflendi\u011finde 30%&#039;ye kadar in vivo d\u00fczenleme verimlili\u011fine ula\u015ft\u0131. NanoCas ayr\u0131ca, SaCas9 ile 10%&#039;ye k\u0131yasla kalp boyunca 15% d\u00fczenleme g\u00f6sterdi. Karaci\u011fer dokusunun analizi, minimal hedef d\u0131\u015f\u0131 d\u00fczenleme g\u00f6sterdi.<\/li>\n<\/ul>","protected":false},"excerpt":{"rendered":"<p>Several research groups have been attempting to create compact CRISPR systems that can fit into a single AAV vector while maintaining editing capabilities. The group at Mammoth Biosciences is one of these; they might have found success. Its researchers started by sorting through genetic sequence information from several CRISPR types that are found in microorganisms. [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":2724,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[66,226,72,227,225,228],"class_list":["post-2723","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-duchenne-muscular-dystrophy","tag-dystrophin-gene","tag-gene-editing","tag-mammoth-biosciences","tag-mini-crispr","tag-nanocas"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/2723","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=2723"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/2723\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/2724"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=2723"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=2723"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=2723"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}