{"id":4151,"date":"2025-07-18T12:17:47","date_gmt":"2025-07-18T09:17:47","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4151"},"modified":"2025-07-18T12:17:49","modified_gmt":"2025-07-18T09:17:49","slug":"uw-medicine-announces-new-gene-therapy-research-for-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/uw-medicine-announces-new-gene-therapy-research-for-duchenne-muscular-dystrophy\/","title":{"rendered":"UW Medicine, Duchenne Kas Distrofisi i\u00e7in Yeni Gen Terapisi Ara\u015ft\u0131rmas\u0131n\u0131 Duyurdu"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Duchenne kas distrofisi (DMD) hastal\u0131\u011f\u0131na yakalananlar i\u00e7in yeni bir gen tedavisi, hem kas kayb\u0131n\u0131 durdurma hem de gelecekte hasarl\u0131 kaslar\u0131 onarma konusunda umut vadediyor. <strong>UW Medicine, tedavinin bir sonraki ad\u0131m\u0131n\u0131n yakla\u015f\u0131k iki y\u0131l i\u00e7inde ba\u015flamas\u0131 beklenen insan denemeleri olaca\u011f\u0131n\u0131 s\u00f6yledi.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">UW Medicine liderli\u011findeki \u00e7al\u0131\u015fman\u0131n amac\u0131, kaslardaki hasarl\u0131 DMD genini, bir mekik vekt\u00f6r\u00fcn\u00fcn i\u00e7ine bir dizi protein paketi yerle\u015ftirerek de\u011fi\u015ftirmektir. Kas distrofisi olan ki\u015filerde bulunmayan distrofin proteini, ek genetik kod taraf\u0131ndan \u00fcretilecektir.<\/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=\"#currently-no-cure-for-dmd\">\u015eu anda DMD i\u00e7in bir tedavi yok<\/a><\/li><li><a href=\"#who-is-dr-jeffrey-chamberlain\">Dr. Jeffrey Chamberlain kimdir?<\/a><\/li><li><a href=\"#what-is-uw-medicines-new-gene-study\">UW Medicine&#039;nin Yeni Gen \u00c7al\u0131\u015fmas\u0131 Nedir?<\/a><\/li><li><a href=\"#human-trials-should-begin-in-approximately-two-years\">\u0130nsan Deneyleri Yakla\u015f\u0131k \u0130ki Y\u0131l \u0130\u00e7inde Ba\u015flayacak<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"currently-no-cure-for-dmd\">\u015eu anda DMD i\u00e7in bir tedavi yok<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Hastal\u0131\u011f\u0131n \u015fu anda bilinen bir tedavisi yok ve mevcut ila\u00e7lar ve tedaviler sadece hastal\u0131\u011f\u0131 yava\u015flat\u0131yor. Ar\u0131zal\u0131 gen X kromozomunda bulundu\u011fundan, bu \u00e7al\u0131\u015fman\u0131n konusu olan Duchenne kas distrofisi hastalar\u0131 tamamen erkektir. Belirtiler d\u00f6rt ya\u015f\u0131nda ba\u015flar ve genellikle 20&#039;li veya 30&#039;lu ya\u015flar\u0131nda vefat ederler.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Duchenne hastal\u0131\u011f\u0131na y\u00f6nelik yeni gen tedavisine ili\u015fkin bulgular Nature dergisinde yay\u0131mland\u0131. <a href=\"https:\/\/www.nature.com\/articles\/s41586-024-07710-8\" target=\"_blank\" rel=\"noreferrer noopener\">Devam\u0131n\u0131 oku<\/a> \u2013<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"who-is-dr-jeffrey-chamberlain\">Dr. Jeffrey Chamberlain kimdir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fman\u0131n ba\u015fyazar\u0131, UW Medicine&#039;de genetik\u00e7i ve n\u00f6rolog olan Dr. Jeffrey Chamberlain, mesleki hayat\u0131n\u0131 kas distrofisi i\u00e7in bir tedavi veya \u00e7are aray\u0131\u015f\u0131na adam\u0131\u015ft\u0131r. Washington \u00dcniversitesi T\u0131p Fak\u00fcltesi&#039;nde McCaw Vakf\u0131 Kas Distrofisi K\u00fcrs\u00fcs\u00fc&#039;n\u00fc ve Wellstone Kas Distrofisi Ara\u015ft\u0131rma Merkezi&#039;nin direkt\u00f6rl\u00fc\u011f\u00fcn\u00fc y\u00fcr\u00fctmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fmay\u0131 Chamberlain laboratuvar\u0131nda molek\u00fcler biyolog olarak g\u00f6rev yapan Dr. Hichem Tasfaout y\u00f6netti.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">D\u00fczeltilmesi gereken genin &quot;do\u011fadaki en b\u00fcy\u00fck gen&quot; olmas\u0131 ger\u00e7e\u011finin, ara\u015ft\u0131rmac\u0131lar\u0131n ilerleme kaydetmesini engelledi\u011fini iddia etti. Kaslara ve genlerine gerekli protein onar\u0131mlar\u0131n\u0131 sa\u011flamak bug\u00fcne kadar imkans\u0131zd\u0131.<\/p>\n\n\n\n<figure class=\"wp-block-embed is-type-video is-provider-youtube wp-block-embed-youtube wp-embed-aspect-16-9 wp-has-aspect-ratio\"><div class=\"wp-block-embed__wrapper\">\n<div class=\"youtube-embed\" data-video_id=\"0Shrd9SW6O0\"><iframe title=\"Kas Distrofisi \u0130\u00e7in Yeni Gen Terapileri\" width=\"696\" height=\"392\" src=\"https:\/\/www.youtube.com\/embed\/0Shrd9SW6O0?feature=oembed&#038;enablejsapi=1\" frameborder=\"0\" allow=\"accelerometer; autoplay; clipboard-write; encrypted-media; gyroscope; picture-in-picture; web-share\" referrerpolicy=\"strict-origin-when-cross-origin\" allowfullscreen><\/iframe><\/div>\n<\/div><\/figure>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-uw-medicines-new-gene-study\">UW Medicine&#039;nin Yeni Gen \u00c7al\u0131\u015fmas\u0131 Nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Vir\u00fcsten \u00fcretilen ve gen tedavilerini insan h\u00fccrelerine ta\u015f\u0131mak i\u00e7in kullan\u0131lan k\u00fc\u00e7\u00fck mekikler olan \u00e7e\u015fitli adeno-ili\u015fkili viral vekt\u00f6rler veya AAV&#039;ler kullan\u0131larak geli\u015ftirilen yeni yakla\u015f\u0131m, fare modelleri \u00fczerinde umut verici sonu\u00e7lar g\u00f6sterdi. <strong>Bu gen terapisi, tek bir AAV yerine bir dizi AAV kullan\u0131yor; kaslardan terap\u00f6tik protein par\u00e7alar\u0131 al\u0131n\u0131yor ve v\u00fccuda gerekli genetik d\u00fczeltmeyi bir araya getirmeye ba\u015flamak i\u00e7in talimatlar yerle\u015ftiriliyor.<\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"human-trials-should-begin-in-approximately-two-years\">\u0130nsan Deneyleri Yakla\u015f\u0131k \u0130ki Y\u0131l \u0130\u00e7inde Ba\u015flayacak<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Chamberlain, tedavinin bir sonraki a\u015famas\u0131n\u0131n insan deneyleri oldu\u011funu ve bunlar\u0131n yakla\u015f\u0131k iki y\u0131l i\u00e7inde ba\u015flamas\u0131 gerekti\u011fini s\u00f6yledi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu yakla\u015f\u0131m, hastal\u0131\u011f\u0131n daha fazla ilerlemesini durdurman\u0131n yan\u0131 s\u0131ra, laboratuvarda distrofiyle ba\u011flant\u0131l\u0131 patolojinin b\u00fcy\u00fck bir k\u0131sm\u0131n\u0131 d\u00fczeltti. Chamberlain ve Tasfaout, bu yakla\u015f\u0131m\u0131n sonunda kas kayb\u0131n\u0131 tersine \u00e7evirebilece\u011fini ve kas dokusunun normal sa\u011fl\u0131\u011f\u0131na kavu\u015fabilece\u011fini umuyor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Chamberlain&#039;e g\u00f6re en son y\u00f6ntem, daha d\u00fc\u015f\u00fck dozlar\u0131n kullan\u0131m\u0131na izin veren yeni bir AAV vekt\u00f6r tipinden de yararlan\u0131yor; bu da \u00f6nceki y\u00f6ntemlerin olumsuz sonu\u00e7lar\u0131n\u0131n bir k\u0131sm\u0131n\u0131 azalt\u0131yor veya ortadan kald\u0131r\u0131yor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Daha fazla bilgi edin: <a href=\"https:\/\/dmdwarrior.com\/tr\/new-gene-therapies-for-duchenne-muscular-dystrophy\/\">Duchenne Musk\u00fcler Distrofisi \u0130\u00e7in Umut Veren Yeni Gen Terapileri<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>For those who suffer from Duchenne muscular dystrophy (DMD), a novel gene therapy holds promise for both stopping muscle loss and maybe restoring the damaged muscle in the future. The next step for the treatment will be human trials, which are expected to begin in about two years, UW Medicine said. The goal of the [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4158,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[61,396],"class_list":["post-4151","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-gene-therapy","tag-uw-medicine"],"subtitle":"UW Medicine, Duchenne kas distrofisi i\u00e7in geli\u015ftirdi\u011fi gen terapisinin umut verici oldu\u011funu belirtiyor. Kas kayb\u0131n\u0131 tersine \u00e7evirebilece\u011fi ve kas dokusunu normal sa\u011fl\u0131\u011f\u0131na kavu\u015fturabilece\u011fi umuluyor.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4151","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=4151"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4151\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4158"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4151"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4151"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4151"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}