{"id":17502,"date":"2022-11-02T07:38:39","date_gmt":"2022-11-02T11:38:39","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?page_id=17502"},"modified":"2023-12-20T05:15:53","modified_gmt":"2023-12-20T10:15:53","slug":"emilys-entourage-awards-220000-to-university-of-iowa-laboratory-to-advance-research-into-correcting-nonsense-mutation-cftr-function","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/tr\/emilys-entourage-awards-220000-to-university-of-iowa-laboratory-to-advance-research-into-correcting-nonsense-mutation-cftr-function\/","title":{"rendered":"Emily's Entourage, Sa\u00e7ma Mutasyon CFTR \u0130\u015flevinin D\u00fczeltilmesine Y\u00f6nelik Ara\u015ft\u0131rmalar\u0131 \u0130lerletmek i\u00e7in Iowa \u00dcniversitesi Laboratuvar\u0131na $220,000 \u00d6d\u00fcl Verdi"},"content":{"rendered":"

Proje, KF'nin nonsense mutasyonlar\u0131na sahip ki\u015filerin hava yollar\u0131ndaki kistik fibroz transmembran iletkenlik reg\u00fclat\u00f6r\u00fc (CFTR) proteinini d\u00fczeltmek i\u00e7in yeni sistemler sunuyor<\/i>
\n<\/strong><\/em><\/h3>\n

2 Kas\u0131m 2022 - Nonsense mutasyonuna sahip olanlar da dahil olmak \u00fczere kistik fibrozis (KF) hastalar\u0131n\u0131n son 10%'si i\u00e7in yeni tedaviler ve tedavi ara\u015ft\u0131rmalar\u0131n\u0131 h\u0131zland\u0131ran yenilik\u00e7i bir 501(c)3 vakf\u0131 olan Emily's Entourage (EE), bug\u00fcn Iowa \u00dcniversitesi'nden Patrick L. Sinn'e, KF'nin en az bir nonsense mutasyonuna sahip ki\u015filerde CFTR proteinini d\u00fczeltme ara\u015ft\u0131rmas\u0131n\u0131 desteklemek i\u00e7in $220.000 fon verdi\u011fini duyurdu.<\/span><\/p>\n

\"\"

Iowa \u00dcniversitesi'nden Patrick L. Sinn, Adenin Baz D\u00fczenleyicisi (ABE) ad\u0131 verilen m\u00fchendislik \u00fcr\u00fcn\u00fc bir enzim kullanarak CFTR mutasyonlar\u0131n\u0131n b\u00f6lgeye \u00f6zg\u00fc onar\u0131m\u0131n\u0131 sa\u011flamay\u0131 ama\u00e7layan bir ara\u015ft\u0131rma y\u00fcr\u00fct\u00fcyor.<\/p><\/div>\n

CFTR fonksiyonu ve h\u00fccre tipi ifadesi hakk\u0131ndaki bilgiler, 1989'daki ke\u015ffinden bu yana b\u00fcy\u00fck \u00f6l\u00e7\u00fcde ilerlemi\u015ftir. \u015eu anda, KF'li ki\u015filerin 90%'si, CFTR mutasyonlar\u0131n\u0131n i\u015flevini geri kazand\u0131ran mutasyon hedefli tedavilerden faydalanmaktad\u0131r. Ancak, KF'li ki\u015filerin geri kalan 10%'si \u00e7ok az i\u015flevsel protein \u00fcrettikleri veya hi\u00e7 \u00fcretmedikleri i\u00e7in bu ila\u00e7lardan yararlanamamaktad\u0131r.<\/span><\/p>\n

\"H\u00fccre d\u0131\u015f\u0131 vezik\u00fclle iletilen baz d\u00fczenleyiciler kullan\u0131larak anlams\u0131z CFTR mutasyonlar\u0131n\u0131n d\u00fczeltilmesi\" ba\u015fl\u0131kl\u0131 proje, Adenin Baz D\u00fczenleyici (ABE) ad\u0131 verilen m\u00fchendislik \u00fcr\u00fcn\u00fc bir enzim kullanarak CFTR mutasyonlar\u0131n\u0131n b\u00f6lgeye \u00f6zg\u00fc onar\u0131m\u0131n\u0131 sa\u011flamay\u0131 ama\u00e7l\u0131yor. Dr. Sinn'in ekibi, ABE'yi terap\u00f6tik olmas\u0131 i\u00e7in uygun hava yolu h\u00fccrelerine yeterince ula\u015ft\u0131rmaya odaklanacak.<\/span><\/p>\n

Emily's Entourage'\u0131n Ba\u015f Bilimsel Sorumlusu Chandra Ghose, \"Dr. Sinn'in laboratuvar\u0131, en son gen d\u00fczenleme teknolojilerini kullanma konusunda kan\u0131tlanm\u0131\u015f bir ge\u00e7mi\u015fe sahiptir\" dedi. \"KF alan\u0131nda muazzam bir ilerleme kaydedilece\u011fini ve nadir ve anlams\u0131z mutasyonlara sahip KF'li ki\u015filerin son 10%'sine \u00f6zellikle dikkat edilerek yeni tedavilerin geli\u015ftirilmesine daha fazla bilgi sa\u011flayaca\u011f\u0131n\u0131 tahmin ediyoruz.<\/span>.\"<\/span><\/p>\n

Proje, v\u00fccuttaki \u00e7o\u011fu h\u00fccrenin \u00fcretti\u011fi h\u00fccre d\u0131\u015f\u0131 vezik\u00fcller (EV'ler) ad\u0131 verilen do\u011fal olarak olu\u015fan par\u00e7ac\u0131klar\u0131 kullanacak. Bu partik\u00fcller \u00e7ok k\u00fc\u00e7\u00fckt\u00fcr ve h\u00fccreler aras\u0131nda k\u00fc\u00e7\u00fck molek\u00fcller ve proteinler ta\u015f\u0131yabilen h\u00fccresel haberciler olarak evrimle\u015fmi\u015ftir. Dr. Sinn'in laboratuvar\u0131 daha \u00f6nce EV'lerin k\u00fc\u00e7\u00fck RNA'lar\u0131 ve proteinleri hava yolu h\u00fccrelerine iletebildi\u011fini g\u00f6stermi\u015ftir. Bu hibenin bir par\u00e7as\u0131 olarak, nadir g\u00f6r\u00fclen, anlams\u0131z CFTR mutasyonlar\u0131n\u0131n \u00e7o\u011funu d\u00fczeltmek i\u00e7in ABE'leri paketleyip iletip iletemeyeceklerini test edecekler.<\/span><\/p>\n

Iowa \u00dcniversitesi Pediatri B\u00f6l\u00fcm\u00fc, Pulmoner T\u0131p Anabilim Dal\u0131'nda profes\u00f6r olan Dr. Sinn, \"Bu yakla\u015f\u0131m\u0131n potansiyeli konusunda \u00e7ok hevesliyim\" dedi. \"Deneylerimiz ba\u015far\u0131l\u0131 olursa, g\u00fc\u00e7l\u00fc bir model sistemde gen d\u00fczenleme deneyleri yapmak i\u00e7in h\u0131zl\u0131 ve ucuz bir tekni\u011fe ve kistik fibroz gibi genetik hastal\u0131klar i\u00e7in potansiyel bir tedaviye sahip olaca\u011f\u0131z. Nihai hedefimiz, bu aerosolize tedavinin tek bir dozundan \u00f6m\u00fcr boyu CFTR gen d\u00fczeltmesi elde etmektir.\"<\/span><\/p>\n

Emily's Entourage'\u0131n finanse etti\u011fi hibeler hakk\u0131nda daha fazla bilgi i\u00e7in <\/span>www.emilysentourage.org\/awarded-grants<\/span><\/a>.\u00a0<\/span><\/p>\n

###<\/span><\/p>\n

Emily'nin \u00c7evresi Hakk\u0131nda<\/b><\/p>\n

Emily's Entourage, KF'nin nonsense mutasyonlar\u0131na sahip olanlar da dahil olmak \u00fczere mevcut mutasyon hedefli tedavilerden fayda g\u00f6rmeyen kistik fibrozis (KF) pop\u00fclasyonunun son 10%'sindeki bireyler i\u00e7in yeni tedaviler ve bir tedavi i\u00e7in ara\u015ft\u0131rmalar\u0131 h\u0131zland\u0131ran yenilik\u00e7i bir 501(c)3't\u00fcr. Emily's Entourage 2011 y\u0131l\u0131ndan bu yana milyonlarca dolarl\u0131k ara\u015ft\u0131rma hibesi vermi\u015f, bir KF gen terapisi \u015firketi kurmu\u015f, klinik deneylere kat\u0131l\u0131m\u0131 h\u0131zland\u0131rmak i\u00e7in bir hasta kay\u0131t defteri ve klinik deney e\u015fle\u015ftirme program\u0131 geli\u015ftirmi\u015f ve d\u00fcnya \u00e7ap\u0131nda y\u00fcksek etkili ara\u015ft\u0131rma ve ila\u00e7 geli\u015ftirme \u00e7abalar\u0131na \u00f6nc\u00fcl\u00fck etmi\u015ftir. Kurulu\u015f, New York Times, STAT, CNN, People ve daha fazlas\u0131 dahil olmak \u00fczere ulusal medyada yer alm\u0131\u015ft\u0131r. Daha fazla bilgi i\u00e7in <\/span>emilysentourage.org<\/span><\/a>.\u00a0<\/span><\/p>\n

Medya \u0130leti\u015fim<\/b>
\nAshling Knight<\/span>
\nPazarlama ve \u0130leti\u015fim Direkt\u00f6r\u00fc<\/span>
\nEmily'nin \u00c7evresi<\/span>
\nashling@emilysentourage.org<\/span><\/a>\u00a0<\/span><\/p>","protected":false},"excerpt":{"rendered":"

Proje, KF'nin sa\u00e7ma mutasyonlar\u0131na sahip ki\u015filerin solunum yollar\u0131ndaki kistik fibroz transmembran iletkenlik reg\u00fclat\u00f6r\u00fc (CFTR) proteinini d\u00fczeltmek i\u00e7in yeni sistemler sunuyor 2 Kas\u0131m 2022 - Kistik fibrozlu (KF) ki\u015filerin son 10%'si i\u00e7in yeni tedaviler ve bir tedavi ara\u015ft\u0131rmas\u0131n\u0131 h\u0131zland\u0131ran yenilik\u00e7i bir 501(c)3 vakf\u0131 olan Emily's Entourage (EE), ... <\/p>\n

<\/div>\n

Daha fazla bilgi edinin<\/a><\/p>","protected":false},"author":1281,"featured_media":16426,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_seopress_robots_primary_cat":"none","_seopress_titles_title":"Emily\u2019s Entourage Awards $220,000 to University of Iowa Laboratory to Advance Research Into Correcting Nonsense Mutation CFTR Function","_seopress_titles_desc":"The project introduces novel systems for correcting the cystic fibrosis transmembrane conductance regulator (CFTR) protein in the airways of people with nonsense mutations of CF.","_seopress_robots_index":"","_monsterinsights_skip_tracking":false,"_monsterinsights_sitenote_active":false,"_monsterinsights_sitenote_note":"","_monsterinsights_sitenote_category":0,"_jetpack_memberships_contains_paid_content":false,"footnotes":""},"categories":[178],"tags":[],"class_list":["post-17502","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-press-releases"],"acf":[],"jetpack_featured_media_url":"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/Emilys-Entourage-Logo_Website-Featured-Image.png","jetpack_sharing_enabled":true,"post_mailing_queue_ids":[],"_links":{"self":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/17502","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/users\/1281"}],"replies":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/comments?post=17502"}],"version-history":[{"count":5,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/17502\/revisions"}],"predecessor-version":[{"id":20533,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/17502\/revisions\/20533"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media\/16426"}],"wp:attachment":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media?parent=17502"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/categories?post=17502"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/tags?post=17502"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}