{"id":3185,"date":"2025-03-12T13:40:19","date_gmt":"2025-03-12T10:40:19","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3185"},"modified":"2025-03-12T13:40:20","modified_gmt":"2025-03-12T10:40:20","slug":"hydrogel-delivery-system-holds-potential-for-cell-based-therapy-of-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/hydrogel-delivery-system-holds-potential-for-cell-based-therapy-of-duchenne-muscular-dystrophy\/","title":{"rendered":"Hidrojel Da\u011f\u0131t\u0131m Sistemi, Duchenne Musk\u00fcler Distrofisinin H\u00fccre Bazl\u0131 Tedavisi \u0130\u00e7in Potansiyel Ta\u015f\u0131yor"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u0130ngiltere&#039;deki ara\u015ft\u0131rmac\u0131lar, kas distrofisi fare modelinde, hidrojeller i\u00e7inde CRISPR ile d\u00fczeltilmi\u015f insan miyojenik h\u00fccrelerinin kullan\u0131larak canl\u0131 kas dokusu \u00fcreten klinik olarak uygulanabilir bir nakil y\u00f6ntemini duyurdular.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Distrofin genindeki mutasyonlar, g\u00fc\u00e7ten d\u00fc\u015f\u00fcren ve tedavi edilemez bir kas erimesi durumu olan Duchenne kas distrofisine (DMD) neden olur. H\u00fccre bazl\u0131 tedavilerin potansiyeline ra\u011fmen, ba\u015far\u0131l\u0131 a\u015f\u0131lama i\u00e7in miyojenik potansiyele sahip yeterli k\u00f6k\/progenitor h\u00fccre elde etmek zordur.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Hidrojel Da\u011f\u0131t\u0131m Sistemi<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Transplantasyon i\u00e7in sonsuz miktarda miyojenik progenitor h\u00fccre (MPC) \u00fcretmek amac\u0131yla, insan pluripotent k\u00f6k h\u00fccreleri, embriyonik k\u00f6k h\u00fccreleri veya ind\u00fcklenmi\u015f pluripotent k\u00f6k h\u00fccreleri i\u00e7in farkl\u0131la\u015fma protokollerindeki geli\u015fmeler sayesinde art\u0131k bu engel a\u015f\u0131labilir. Ancak, don\u00f6r h\u00fccrelerinin intram\u00fcsk\u00fcler enjeksiyonu, etkilenen iskelet kas\u0131n\u0131n geni\u015f b\u00f6lgelerini kaplamak i\u00e7in y\u00fczlerce enjeksiyon gerekece\u011finden klinikte invaziv ve pratik olmayan bir uygulama olarak kabul edilir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Londra Queen Mary \u00dcniversitesi&#039;ndeki ara\u015ft\u0131rmac\u0131lar, Dr. Yung-Yao Lin y\u00f6netiminde, bu iletim zorlu\u011funun \u00fcstesinden gelmek i\u00e7in, DMD hastas\u0131 k\u00f6k h\u00fccrelerinden t\u00fcretilen CRISPR d\u00fczeltilmi\u015f insan miyojenik progenit\u00f6r h\u00fccreleri (MPC&#039;ler) ile hidrojel arac\u0131l\u0131 iletimi birle\u015ftiren bir nakil stratejisi geli\u015ftirdiler. <strong>Bulgular\u0131 yak\u0131n zamanda Cell Reports Medicine&#039;de yay\u0131mland\u0131.<\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Tam uzunlukta distrofin<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Blizard Enstit\u00fcs\u00fc ve UCL Great Ormond Street \u00c7ocuk Sa\u011fl\u0131\u011f\u0131 Enstit\u00fcs\u00fc&#039;n\u00fcn i\u015fbirli\u011fiyle y\u00fcr\u00fct\u00fclen \u00e7al\u0131\u015fmada, \u00fcretilen 3 boyutlu h\u00fccre y\u00fckl\u00fc hidrojel yap\u0131lar\u0131n, konak kas \u00f6n i\u015flemine gerek kalmadan distrofin eksikli\u011fi olan mdx \u00e7\u0131plak farelere ba\u015far\u0131yla a\u015f\u0131land\u0131\u011f\u0131 g\u00f6sterildi. <strong>Ekip, nakledilen h\u00fccrelerde tam uzunlukta distrofinin, nakilden 4 hafta ve 5-6 ay sonra olu\u015ftu\u011funu s\u00f6yledi.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">En \u00f6nemli bulgu, insan miyofibrlerinin distrofik fare kas\u0131na i\u015flevsel olarak entegre olmas\u0131, fare motor n\u00f6ronlar\u0131yla n\u00f6rom\u00fcsk\u00fcler ba\u011flant\u0131lar kurmas\u0131 ve fare damar sistemi taraf\u0131ndan s\u00fcrd\u00fcr\u00fclmesiydi. Ek olarak, insan PAX7 pozitif h\u00fccreleri uydu h\u00fccre ni\u015fini doldurdu ve bu da s\u00fcrekli kas rejenerasyonu olas\u0131l\u0131\u011f\u0131n\u0131 g\u00f6sterdi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Neyse ki, \u00e7al\u0131\u015fma uzun s\u00fcreli tutunma (5-6 ay) olan farelerde kanser belirtisine rastlanmamas\u0131yla \u00f6nemli g\u00fcvenlik endi\u015felerini giderdi. <strong>DMD ve di\u011fer kas kayb\u0131na yol a\u00e7an hastal\u0131klar a\u00e7\u0131s\u0131ndan bu sonu\u00e7lar, hidrojel bazl\u0131 h\u00fccre tedavisi i\u00e7in g\u00fc\u00e7l\u00fc bir kavram kan\u0131t\u0131 sunmaktad\u0131r.<\/strong><\/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>","protected":false},"excerpt":{"rendered":"<p>In a mouse model of muscular dystrophy, researchers in the UK announced a clinically applicable transplantation method that uses CRISPR-corrected human myogenic cells in hydrogels to produce viable muscle tissue. Mutations in the dystrophin gene cause Duchenne muscular dystrophy (DMD), a debilitating and incurable muscle-wasting condition. Despite the potential of cell-based therapies, it is difficult [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3187,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[72,61,259],"class_list":["post-3185","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-gene-editing","tag-gene-therapy","tag-stem-cell-therapy"],"subtitle":"Bu \u00e7al\u0131\u015fma, DMD ve di\u011fer kas kayb\u0131 bozukluklar\u0131 i\u00e7in potansiyel bir tedavi olarak hidrojel bazl\u0131 h\u00fccre terapisine dair ikna edici bir kavram kan\u0131t\u0131 sunmaktad\u0131r.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3185","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=3185"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3185\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3187"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3185"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3185"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3185"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}