Indiana \u00dcniversitesi'nde pediatri profes\u00f6r\u00fc olan Dr. Renzhi Han, Duchenne (DMD) hastal\u0131\u011f\u0131n\u0131n tedavisi i\u00e7in yeni bir gen tedavisi \u00fczerinde \u00e7al\u0131\u015f\u0131yor ve \u00e7al\u0131\u015fman\u0131n devam\u0131 i\u00e7in fon ar\u0131yor.<\/p>\n\n\n\n
Indiana \u00dcniversitesi'nin Temmuz 2024'te yapt\u0131\u011f\u0131 bir a\u00e7\u0131klamaya g\u00f6re, bu yeni gen terapisi fareler \u00fczerinde test edilmi\u015f ve \u00e7ok ba\u015far\u0131l\u0131 sonu\u00e7lar elde edilmi\u015ftir. DMD Warrior olarak, Dr. Renzhi Han ile ileti\u015fime ge\u00e7tik ve bu \u00e7al\u0131\u015fman\u0131n mevcut durumu hakk\u0131nda bilgi ald\u0131k.<\/p>\n\n\n\n
Yo\u011fun \u00e7al\u0131\u015fmalar\u0131n\u0131n aras\u0131nda bize 'Yeni gen terapisi yakla\u015f\u0131m\u0131m\u0131z klinik \u00f6ncesi hayvan modellerinde \u00fcmit verici sonu\u00e7lar g\u00f6sterdi. Yakla\u015f\u0131k iki y\u0131l i\u00e7inde klinik \u00e7al\u0131\u015fmalara ba\u015flamadan \u00f6nce daha fazla klinik \u00f6ncesi etkinlik ve g\u00fcvenlik testi yapmak i\u00e7in fon ar\u0131yoruz.' diye yan\u0131tlad\u0131.<\/p>\n\n\n\n
\u00c7al\u0131\u015fman\u0131n k\u0131demli yazar\u0131 ve IU T\u0131p Fak\u00fcltesi'nde pediatri profes\u00f6r\u00fc olan Dr. Renzhi Han, "Duchenne kas distrofisi i\u00e7in mevcut gen terapisi, distrofinin k\u0131salt\u0131lm\u0131\u015f bir versiyonunu kullan\u0131yor" dedi. "Ne yaz\u0131k ki, bu se\u00e7enek kaslar\u0131 tam olarak korumuyor \u00e7\u00fcnk\u00fc tam uzunluktaki distrofinin bir\u00e7ok \u00f6nemli i\u015flevsel alan\u0131ndan yoksun."<\/p>\n\n\n\n
ABD G\u0131da ve \u0130la\u00e7 Dairesi'nin yak\u0131n zamanda Duchenne kas distrofisi i\u00e7in mikro-distrofin gen terapisini onaylad\u0131\u011f\u0131n\u0131 belirten Han, tedavi sonu\u00e7lar\u0131n\u0131n beklenenden daha az tatmin edici oldu\u011funu s\u00f6yledi.<\/p>\n\n\n\n
Herman B Wells Pediatrik Ara\u015ft\u0131rma Merkezi'ndeki Han ve ekibi, adeno-ili\u015fkili vir\u00fcs y\u00f6ntemlerini kullanarak h\u00fccrelere ekstra b\u00fcy\u00fck terap\u00f6tik genler ula\u015ft\u0131rma deneyimlerine dayanarak, distrofin proteininin tam bir versiyonunu kaslara ula\u015ft\u0131rmak i\u00e7in \u00fc\u00e7l\u00fc adeno-ili\u015fkili bir vir\u00fcs vekt\u00f6r sistemi geli\u015ftirdiler.<\/p>\n\n\n\n
\u201cHan, "Tam uzunluktaki distrofin proteinini etkili bir \u015fekilde \u00fcretip birle\u015ftirebildi\u011finden emin olmak i\u00e7in yeni \u00fc\u00e7 vekt\u00f6rl\u00fc sistemimizi optimize ettik ve test ettik," dedi. \u201c"Verilerimiz, DMD'li farelerin hem iskelet hem de kalp kaslar\u0131nda tam uzunlukta distrofinin ba\u015far\u0131l\u0131 bir \u015fekilde geri kazand\u0131r\u0131ld\u0131\u011f\u0131n\u0131 ve bunun da kas sa\u011fl\u0131klar\u0131nda, g\u00fc\u00e7lerinde ve fonksiyonlar\u0131nda \u00f6nemli iyile\u015fmelere yol a\u00e7t\u0131\u011f\u0131n\u0131 do\u011frulad\u0131."\u201d<\/strong><\/p>\n\n\n\n Han, \u00fc\u00e7l\u00fc adeno ili\u015fkili vir\u00fcs vekt\u00f6r sistemi i\u00e7in ge\u00e7ici bir patent ba\u015fvurusunda bulundu ve tedaviyi pazara sunmak i\u00e7in IU \u0130novasyon ve Ticarile\u015ftirme Ofisi ile i\u015f birli\u011fi yap\u0131yor. Ayr\u0131ca Duchenne kas distrofisi hastalar\u0131n\u0131n umut vadeden yeni tedavi se\u00e7eneklerine eri\u015febilmesi i\u00e7in ek fon ar\u0131yor.<\/p>\n\n\n\n "Bu yeni gen tedavisi yakla\u015f\u0131m\u0131n\u0131n, hastalara \u015fu anda sahip olduklar\u0131 tedavilere k\u0131yasla \u00f6nemli avantajlar sa\u011flad\u0131\u011f\u0131na inan\u0131yorum ve bunu daha ileri klinik geli\u015ftirmeye sunmak i\u00e7in sab\u0131rs\u0131zlan\u0131yorum" dedi.<\/p>\n\n\n\n IU T\u0131p Fak\u00fcltesi'ndeki di\u011fer \u00e7al\u0131\u015fma yazarlar\u0131 aras\u0131nda Yuan Zhou, Chen Zhang, Weidong Xiao ve Roland W. Herzog yer al\u0131yor.<\/p>","protected":false},"excerpt":{"rendered":" Dr. Renzhi Han, a professor of pediatrics at Indiana University, is working on a new gene therapy for the treatment of Duchenne (DMD) and is seeking funding for the continuation of the study. According to an announcement made by Indiana University in July 2024, this new gene therapy has been tested on mice and very […]<\/p>\n","protected":false},"author":1,"featured_media":1399,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[359,895,894],"class_list":["post-1398","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-full-length-dystrophin","tag-indiana-university","tag-renzhi-han"],"subtitle":"","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/1398","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=1398"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/1398\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/1399"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=1398"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=1398"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=1398"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}