{"id":11092,"date":"2018-07-11T09:44:51","date_gmt":"2018-07-11T13:44:51","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?p=11092"},"modified":"2019-11-14T10:04:57","modified_gmt":"2019-11-14T15:04:57","slug":"ee-awards-660k-to-cystic-fibrosis-nonsense-mutations-research","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/tr\/ee-awards-660k-to-cystic-fibrosis-nonsense-mutations-research\/","title":{"rendered":"EE, Kistik Fibrozis Sa\u00e7ma Mutasyonlar Ara\u015ft\u0131rmas\u0131na $660K \u00d6d\u00fcl\u00fc Verdi"},"content":{"rendered":"

Emily's Entourage (EE), uluslararas\u0131 ara\u015ft\u0131rmac\u0131lardan olu\u015fan sayg\u0131n bir gruba iki y\u0131ll\u0131k bir s\u00fcre i\u00e7inde toplam $660.000 tutar\u0131nda \u00fc\u00e7 ara\u015ft\u0131rma hibesi verildi\u011fini duyurmaktan heyecan duymaktad\u0131r. Bu hibeler, Kistik Fibrozis (KF) nonsense mutasyonlar\u0131na sahip ki\u015filer i\u00e7in terap\u00f6tik geli\u015fimi h\u0131zland\u0131rmak amac\u0131yla kritik ara\u015ft\u0131rmalar\u0131 ilerletmektedir.<\/p>\n

\"Bu yeni hibeler, Emily's Entourage'\u0131n KF i\u00e7in yeni terap\u00f6tik geli\u015fimin s\u0131n\u0131rlar\u0131n\u0131 zorlama ve bu at\u0131l\u0131mlar\u0131 hastalara h\u0131zl\u0131 bir \u015fekilde ula\u015ft\u0131rma taahh\u00fcd\u00fcn\u00fc yans\u0131tarak, KF nonsense mutasyonlar\u0131na y\u00f6nelik mevcut ve ara\u015ft\u0131rma ama\u00e7l\u0131 tedavilerin etkinli\u011fini de\u011ferlendirmektedir.\"<\/p>\n

-\u00c7EE Bilimsel Direkt\u00f6r\u00fc Peter Haggie, PhD<\/p><\/blockquote>\n

\n

\"\"W1282X Erken Sonlanma Kodon Mutasyonuna Sahip KF Hastalar\u0131n\u0131n Tedavisinde Yeni Terap\u00f6tik Yakla\u015f\u0131mlar<\/strong><\/h3>\n

Venkateshwar Mutyam, PhD ve Steven M. Rowe, MD, MSPH<\/strong><\/p>\n

Birmingham Alabama \u00dcniversitesi<\/em><\/p>\n

Emily's Entourage taraf\u0131ndan desteklenen \u00f6nceki \u00e7al\u0131\u015fmalar, KALYDECO'nun W1282X mutasyonuna sahip baz\u0131 KF deneklerinde terap\u00f6tik fayda sa\u011flad\u0131\u011f\u0131n\u0131 ortaya koymu\u015ftur. Bu provokatif \u00e7al\u0131\u015fmalar, klinik faydalar\u0131n onayl\u0131 bir d\u00fczeltici-potansiyat\u00f6r tedavi ile daha da art\u0131r\u0131l\u0131p art\u0131r\u0131lamayaca\u011f\u0131n\u0131 de\u011ferlendirmek i\u00e7in n-of-1 klinik \u00e7al\u0131\u015fmalarda geni\u015fletilecektir.<\/p>\n

Strateji: K\u00fct\u00fcphanede Gezinme: Terap\u00f6tik Molek\u00fcllerin Belirlenmesi ve Yeniden Tasarlanmas\u0131<\/em><\/p>\n

 <\/p>\n

 <\/p>\n

\"\"W1282X CFTR Hava Yolu Epitel H\u00fccre Fonksiyonunun K\u00fc\u00e7\u00fck Molek\u00fcllerle \u0130yile\u015ftirilmesi<\/strong><\/h3>\n

Theo Moraes, MD, PhD, Tanja Gonska, MD, Christine Bear, PhD ve Felix Ratjen, MD, PhD, FRCPC<\/strong><\/p>\n

Hasta \u00c7ocuklar Hastanesi, Toronto, Kanada<\/em><\/p>\n

H\u00fccre k\u00fclt\u00fcr\u00fc, CFTR fonksiyonel de\u011ferlendirmesi, terap\u00f6tik geli\u015ftirme ve klinik uygulamalardaki uzmanl\u0131\u011f\u0131 birle\u015ftiren bu proje, mevcut terap\u00f6tik yakla\u015f\u0131mlar\u0131n W1282X mutasyonuna sahip KF deneklerinden t\u00fcretilen hava yolu epitel h\u00fccrelerinde iyon ta\u015f\u0131nmas\u0131 ve mukosiliyer klirens dahil olmak \u00fczere temel \u00f6zellikleri mod\u00fcle edip etmedi\u011fini de\u011ferlendirecektir.<\/p>\n

Strateji: K\u00fct\u00fcphanede Gezinme: Terap\u00f6tik Molek\u00fcllerin Belirlenmesi ve Yeniden Tasarlanmas\u0131<\/em><\/p>\n

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 <\/p>\n

 <\/p>\n

\"\"Nonsense Mutasyonlar\u0131n\u0131n Neden Oldu\u011fu KF'nin CFTR'den Ba\u011f\u0131ms\u0131z Tedavisi \u0130\u00e7in Molek\u00fcler Bir Protez<\/strong><\/h3>\n

Martin D. Burke, MD, PhD ve Michael J. Welsh, MD<\/strong><\/p>\n

Urbana-Champaign'deki Illinois \u00dcniversitesi ve Iowa \u00dcniversitesi<\/em><\/p>\n

KF, CFTR iyon kanal\u0131n\u0131n i\u015flev kayb\u0131ndan kaynaklan\u0131r. CFTR'den ba\u011f\u0131ms\u0131z olarak eksik kanal i\u015flevini geri kazanman\u0131n alternatif yollar\u0131n\u0131n geli\u015ftirilmesi acil kar\u015f\u0131lanmam\u0131\u015f bir t\u0131bbi ihtiya\u00e7t\u0131r. H\u00fccre k\u00fclt\u00fcr\u00fc modellerinde ve KF hayvan modellerinde yap\u0131lan zorlay\u0131c\u0131 \u00e7al\u0131\u015fmalara dayanan bu proje, bu ihtiyac\u0131 do\u011frudan ele almak i\u00e7in yeni bir terap\u00f6tik stratejiyi test edecektir. Bu yakla\u015f\u0131m, alternatif bir endikasyon i\u00e7in onaylanm\u0131\u015f bir ilac\u0131 kullan\u0131r ve sonunda KF i\u00e7in yeni bir terap\u00f6tik yakla\u015f\u0131m\u0131n geli\u015ftirilmesine yol a\u00e7abilir.<\/p>\n

Strateji: Yarat\u0131c\u0131 Ge\u00e7ici \u00c7\u00f6z\u00fcmler: Alternatif Yollarla \u00c7al\u0131\u015fma<\/em><\/p>\n

 <\/p>\n

\n

Bu hibeler, Avrupa Komisyonu taraf\u0131ndan desteklenen ikinci tur finansman\u0131 temsil etmektedir. Catalyst for a Cure Kampanyas\u0131<\/strong><\/em>EE Bilimsel Dan\u0131\u015fma Kurulu taraf\u0131ndan KF sa\u00e7ma mutasyonlar\u0131 \u00fczerine ara\u015ft\u0131rma ve ila\u00e7 geli\u015ftirmeyi h\u0131zland\u0131rmak i\u00e7in belirlenen stratejik ara\u015ft\u0131rma alanlar\u0131n\u0131 finanse etmek \u00fczere 2017 y\u0131l\u0131nda ba\u015flat\u0131lan \u00e7\u0131\u011f\u0131r a\u00e7an $3 milyonluk bir giri\u015fim.<\/p>\n

Bunun Nas\u0131l Bir Etkisi Olacak?<\/strong><\/h3>\n

Emily's Entourage, KF nonsense mutasyonu olan hastalara h\u0131zl\u0131 bir \u015fekilde ula\u015fabilecek hayat kurtar\u0131c\u0131 tedavilerin geli\u015ftirilmesini h\u0131zland\u0131rmak i\u00e7in 2011 y\u0131l\u0131ndan bu yana $3.3 milyondan fazla \u00f6d\u00fcl vermi\u015ftir. Sa\u00e7ma mutasyonun iki kopyas\u0131na sahip hastalar, \u015fu anda hedefe y\u00f6nelik tedavilerin bulunmad\u0131\u011f\u0131 KF pop\u00fclasyonunun d\u0131\u015fta kalan 10%'sinin en b\u00fcy\u00fck b\u00f6l\u00fcm\u00fcn\u00fc temsil etmektedir.<\/p>\n

KF nonsense mutasyonlar\u0131 i\u00e7in yenilik\u00e7i tedavi yakla\u015f\u0131mlar\u0131 geli\u015ftirerek \u00f6nc\u00fcl\u00fck eden kararl\u0131 bilim insanlar\u0131na ve bu \u00f6nc\u00fc \u00e7abalar\u0131 destekleyen inan\u0131lmaz destek\u00e7ilerimize i\u00e7tenlikle te\u015fekk\u00fcr etmek istiyoruz.<\/p>\n

Daha fazla bilgi edinmek i\u00e7in \u00d6d\u00fcllendirilen Hibeler<\/a> Sayfa.<\/p>","protected":false},"excerpt":{"rendered":"

Emily\u2019s Entourage (EE) is thrilled to announce that three research grants have been awarded to an esteemed group of international researchers, amounting to a total of $660,000 to be given over a two-year period. These grants advance critical research with the singular goal of expediting therapeutic development for people with Cystic Fibrosis (CF) nonsense mutations…. <\/p>\n

<\/div>\n

Daha fazla bilgi edinin<\/a><\/p>","protected":false},"author":58,"featured_media":10320,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_seopress_titles_title":"","_seopress_titles_desc":"","_seopress_robots_index":"","_seopress_robots_follow":"","_seopress_robots_imageindex":"","_seopress_robots_snippet":"","_seopress_robots_primary_cat":"none","_seopress_robots_breadcrumbs":"","_seopress_robots_freeze_modified_date":"","_seopress_robots_custom_modified_date":"","_seopress_robots_canonical":"","_seopress_social_fb_title":"","_seopress_social_fb_desc":"","_seopress_social_fb_img":"","_seopress_social_fb_img_attachment_id":0,"_seopress_social_fb_img_width":0,"_seopress_social_fb_img_height":0,"_seopress_social_twitter_title":"","_seopress_social_twitter_desc":"","_seopress_social_twitter_img":"","_seopress_social_twitter_img_attachment_id":0,"_seopress_social_twitter_img_width":0,"_seopress_social_twitter_img_height":0,"_seopress_redirections_value":"","_seopress_redirections_enabled":"","_seopress_redirections_enabled_regex":"","_seopress_redirections_logged_status":"","_seopress_redirections_param":"","_seopress_redirections_type":301,"_seopress_analysis_target_kw":"","_seopress_news_disabled":"","_seopress_video_disabled":"","_seopress_video":[{"url":"","title":"","desc":"","thumbnail":"","duration":"","rating":"","view_count":"","tag":"","cat":""}],"_seopress_pro_schemas_manual":null,"_seopress_pro_rich_snippets_disable_all":"","_seopress_pro_rich_snippets_disable":[],"_seopress_pro_schemas":[],"_monsterinsights_skip_tracking":false,"_monsterinsights_sitenote_active":false,"_monsterinsights_sitenote_note":"","_monsterinsights_sitenote_category":0,"_jetpack_newsletter_access":"","_jetpack_dont_email_post_to_subs":false,"_jetpack_newsletter_tier_id":0,"_jetpack_memberships_contains_paywalled_content":false,"_jetpack_feature_clip_id":0,"_jetpack_memberships_contains_paid_content":false,"footnotes":"","jetpack_post_was_ever_published":false},"categories":[1],"tags":[],"class_list":["post-11092","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-uncategorized"],"acf":[],"jetpack_featured_media_url":"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/IMG_0652.jpg","jetpack_sharing_enabled":true,"post_mailing_queue_ids":[],"_links":{"self":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/11092","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\/58"}],"replies":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/comments?post=11092"}],"version-history":[{"count":5,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/11092\/revisions"}],"predecessor-version":[{"id":13807,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/11092\/revisions\/13807"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media\/10320"}],"wp:attachment":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media?parent=11092"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/categories?post=11092"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/tags?post=11092"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}