{"id":11486,"date":"2018-09-07T15:21:46","date_gmt":"2018-09-07T19:21:46","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?p=11486"},"modified":"2018-09-07T15:30:23","modified_gmt":"2018-09-07T19:30:23","slug":"therapeutic-development-for-nonsense-mutations-the-final-frontier-of-cf","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/tr\/therapeutic-development-for-nonsense-mutations-the-final-frontier-of-cf\/","title":{"rendered":"Sa\u00e7ma Mutasyonlar i\u00e7in Terap\u00f6tik Geli\u015ftirme: KF'nin Son S\u0131n\u0131r\u0131"},"content":{"rendered":"<p>Emily's Entourage (EE) \u00fc\u00e7\u00fcnc\u00fc bilimsel sempozyumunu duyuruyor, <em>Sa\u00e7ma Mutasyonlar i\u00e7in Terap\u00f6tik Geli\u015ftirme: KF'nin Son S\u0131n\u0131r\u0131<\/em>24-25 Eyl\u00fcl tarihlerinde Philadelphia, PA'daki Wistar Enstit\u00fcs\u00fc'nde ger\u00e7ekle\u015ftirilecektir. Birincil odak noktas\u0131, hedefe y\u00f6nelik bir tedavi olmaks\u0131z\u0131n kistik fibrozis (KF) pop\u00fclasyonunun son 10%'si i\u00e7in at\u0131l\u0131mlar\u0131 ve tedaviyi h\u0131zland\u0131rmak olacakt\u0131r.<\/p>\n<div id=\"attachment_11501\" style=\"width: 910px\" class=\"wp-caption aligncenter\"><img fetchpriority=\"high\" decoding=\"async\" aria-describedby=\"caption-attachment-11501\" class=\"wp-image-11501 size-full\" src=\"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/WistarInstitute.jpg\" alt=\"\" width=\"900\" height=\"563\" srcset=\"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/WistarInstitute.jpg 900w, https:\/\/www.emilysentourage.org\/wp-content\/uploads\/WistarInstitute-300x188.jpg 300w, https:\/\/www.emilysentourage.org\/wp-content\/uploads\/WistarInstitute-768x480.jpg 768w, https:\/\/www.emilysentourage.org\/wp-content\/uploads\/WistarInstitute-600x375.jpg 600w\" sizes=\"(max-width: 900px) 100vw, 900px\" \/><p id=\"caption-attachment-11501\" class=\"wp-caption-text\">Philadelphia, PA'daki Wistar Enstit\u00fcs\u00fc<\/p><\/div>\n<p>D\u00fcnyan\u0131n d\u00f6rt bir yan\u0131ndan \u00f6nde gelen KF ara\u015ft\u0131rmac\u0131lar\u0131 ve \u015firketlerinin davet edildi\u011fi bu toplant\u0131, KF sa\u00e7ma mutasyonlar\u0131na y\u00f6nelik hayat kurtar\u0131c\u0131 terap\u00f6tiklerin geli\u015ftirilmesini h\u0131zland\u0131rmak i\u00e7in i\u015fbirli\u011fini, fikir al\u0131\u015fveri\u015fini ve yarat\u0131c\u0131 problem \u00e7\u00f6zmeyi destekleyen samimi bir etkinlik olacakt\u0131r. Terap\u00f6tik geli\u015fimin bu alan\u0131 \u00f6zellikle \u00f6nemlidir \u00e7\u00fcnk\u00fc oyunun kurallar\u0131n\u0131 de\u011fi\u015ftiren CFTR mod\u00fcle edici tedaviler \u015fu anda KF pop\u00fclasyonunun yakla\u015f\u0131k 90%'si i\u00e7in onaylanm\u0131\u015f veya ge\u00e7 a\u015fama klinik geli\u015fim a\u015famas\u0131ndad\u0131r. KF sa\u00e7ma mutasyonlar\u0131na sahip olanlar, onaylanm\u0131\u015f veya son a\u015fama CFTR mod\u00fcle edici tedavisi olmayan KF pop\u00fclasyonunun en b\u00fcy\u00fck b\u00f6l\u00fcm\u00fcn\u00fc temsil etmektedir.<\/p>\n<blockquote><p>\"Emily's Entourage'da, KF toplulu\u011fu i\u00e7in terap\u00f6tik geli\u015fimde b\u00fcy\u00fck bir bo\u015flu\u011fu temsil eden sa\u00e7ma mutasyonlarla kolektif olarak m\u00fccadele etmek i\u00e7in en iyi ve en parlaklar\u0131 bir araya getirmekten inan\u0131lmaz heyecan duyuyoruz\" - Emily Kramer-Golinkoff, EE Kurucu Orta\u011f\u0131<\/p><\/blockquote>\n<p>Sempozyum, toplant\u0131dan bir gece \u00f6nce konuklar\u0131n EE Bilimsel Dan\u0131\u015fma Kurulu Ba\u015fkan\u0131 Kevin Foskett, PhD, Bilimsel Direkt\u00f6r Pete Haggie, PhD ve Emily taraf\u0131ndan s\u0131cak bir \u015fekilde kar\u015f\u0131lanaca\u011f\u0131 bir ho\u015f geldiniz yeme\u011fi ile ba\u015flayacakt\u0131r. Ak\u015fam yeme\u011finin amac\u0131, kayna\u015fmak, a\u011f kurmak ve zorlu, verimli \u00e7al\u0131\u015fman\u0131n ger\u00e7ekle\u015fece\u011fi ertesi g\u00fcn i\u00e7in hedefleri dile getirmek olacakt\u0131r.<\/p>\n<div id=\"attachment_11502\" style=\"width: 910px\" class=\"wp-caption aligncenter\"><img decoding=\"async\" aria-describedby=\"caption-attachment-11502\" class=\"wp-image-11502 size-full\" src=\"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/EE-Sep2015.jpg\" alt=\"\" width=\"900\" height=\"563\" srcset=\"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/EE-Sep2015.jpg 900w, https:\/\/www.emilysentourage.org\/wp-content\/uploads\/EE-Sep2015-300x188.jpg 300w, https:\/\/www.emilysentourage.org\/wp-content\/uploads\/EE-Sep2015-768x480.jpg 768w, https:\/\/www.emilysentourage.org\/wp-content\/uploads\/EE-Sep2015-600x375.jpg 600w\" sizes=\"(max-width: 900px) 100vw, 900px\" \/><p id=\"caption-attachment-11502\" class=\"wp-caption-text\">EE'nin Eyl\u00fcl 2015'teki Kilit Fikir \u00d6nderleri toplant\u0131s\u0131<\/p><\/div>\n<p>25 Eyl\u00fcl'de Emily a\u00e7\u0131l\u0131\u015f konu\u015fmas\u0131n\u0131 yapacak, insanlara Emily's Entourage'\u0131 ve misyonunu tan\u0131tacak, toplant\u0131n\u0131n hedeflerine ve aciliyetine insani bir y\u00fcz kazand\u0131racak. Bunu takip edecek toplant\u0131, her biri k\u0131sa sunumlar ve tart\u0131\u015fmalardan olu\u015fan d\u00f6rt ana oturuma b\u00f6l\u00fcnecektir. Bu d\u00f6rt oturum \u015funlard\u0131r:<\/p>\n<p><strong>I. CFTR Mod\u00fclat\u00f6rleri: Kesilmi\u015f CFTR'ler i\u00e7in mod\u00fclat\u00f6rlerin geli\u015ftirilmesi, uyarlanmas\u0131 ve test edilmesi<\/strong><\/p>\n<p style=\"padding-left: 30px;\"><em>Oturum Ba\u015fkan\u0131 Pete Haggie, PhD (University of California, San Francisco, Emily's Entourage)<\/em><\/p>\n<p><strong>II. CFTR Gen tedavisi: Faz I denemelerine ilerleme<\/strong><\/p>\n<p style=\"padding-left: 30px;\"><em>Oturum Ba\u015fkan\u0131 Beverly Davidson, PhD (Philadelphia \u00c7ocuk Hastanesi, Pennsylvania \u00dcniversitesi, Spark Therapeutics, Talee Bio)<\/em><\/p>\n<p><strong>III. N\u00fckleik asit tedavileri: mRNA, tRNA ve oligon\u00fckleotid hedefli yakla\u015f\u0131mlar\u0131n sunulmas\u0131<\/strong><\/p>\n<p style=\"padding-left: 30px;\"><em>Oturum Ba\u015fkanlar\u0131: Phil Thomas (Teksas \u00dcniversitesi, G\u00fcneybat\u0131) ve Aliasger Salem (Iowa \u00dcniversitesi)<\/em><\/p>\n<p><strong>IV. Zaman kazanmak: Alternatif hedefler ve yakla\u015f\u0131mlar<\/strong><\/p>\n<p style=\"padding-left: 30px;\"><em>Oturum Ba\u015fkan\u0131 Kevin Foskett, PhD (Pennsylvania \u00dcniversitesi, Emily's Entourage)<\/em><\/p>\n<p>Sunum yapanlar\u0131n listesi:<\/p>\n<ul>\n<li>Alan Verkman, MD, PhD (Kaliforniya \u00dcniversitesi, San Francisco)<\/li>\n<li>Neal Sharpe, PhD (Eloxx Pharmaceuticals)<\/li>\n<li>Jeffrey Beekman, PhD (\u00dcniversite T\u0131p Merkezi, Utrecht, Hollanda)<\/li>\n<li>Steven M. Rowe, MD, MSPH (Birmingham'daki Alabama \u00dcniversitesi)<\/li>\n<li>Paul McCray, MD (Iowa \u00dcniversitesi)<\/li>\n<li>Patrick Harrison, PhD (University College Cork, \u0130rlanda)<\/li>\n<li>Christopher Ahern, PhD (Iowa \u00dcniversitesi)<\/li>\n<li>Lulu Huang, PhD (Ionis Pharmaceuticals)<\/li>\n<li>James Dahlman, PhD (Georgia Tech)<\/li>\n<li>John Miller, PhD (Proteostasis Therapeutics)<\/li>\n<li>Robert Tarran, PhD (Chapel Hill'deki North Carolina \u00dcniversitesi, Spyryx Biosciences)<\/li>\n<li>Oren Rosenberg, MD, PhD (Kaliforniya \u00dcniversitesi, San Francisco)<\/li>\n<li>Barbara White, MD (Corbus Pharmaceuticals)<\/li>\n<li>Martin D. Burke, MD, PhD (Illinois \u00dcniversitesi Urbana-Champaign)<\/li>\n<\/ul>\n<p>Emily's Entourage, destekleriyle bu toplant\u0131y\u0131 m\u00fcmk\u00fcn k\u0131lan a\u015fa\u011f\u0131daki sponsorlara te\u015fekk\u00fcr eder:<\/p>\n<p><img decoding=\"async\" class=\"size-full wp-image-11521 aligncenter\" src=\"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/2018_EE_Symposium_sponsors.jpg\" alt=\"\" width=\"898\" height=\"420\" srcset=\"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/2018_EE_Symposium_sponsors.jpg 898w, https:\/\/www.emilysentourage.org\/wp-content\/uploads\/2018_EE_Symposium_sponsors-300x140.jpg 300w, https:\/\/www.emilysentourage.org\/wp-content\/uploads\/2018_EE_Symposium_sponsors-768x359.jpg 768w, https:\/\/www.emilysentourage.org\/wp-content\/uploads\/2018_EE_Symposium_sponsors-600x281.jpg 600w\" sizes=\"(max-width: 898px) 100vw, 898px\" \/><span style=\"font-weight: 400;\">Emily's Entourage olarak t\u00fcm sempozyum konu\u015fmac\u0131lar\u0131na, kat\u0131l\u0131mc\u0131lara ve sponsorlara en i\u00e7ten \u015f\u00fckranlar\u0131m\u0131z\u0131 sunar\u0131z. KF sa\u00e7ma mutasyonlar\u0131 i\u00e7in terap\u00f6tik geli\u015fimi h\u0131zland\u0131rmaya hizmet eden sempozyumdan kaynaklanan heyecan verici yeni projeleri ve i\u015fbirliklerini d\u00f6rt g\u00f6zle bekliyoruz. <\/span><\/p>","protected":false},"excerpt":{"rendered":"<p>Emily\u2019s Entourage (EE) announces its third scientific symposium, Therapeutic Development for Nonsense Mutations: The Final Frontier of CF, to be held September 24-25 at The Wistar Institute in Philadelphia, PA. The primary focus will be to accelerate breakthroughs and a cure for the final 10% of the cystic fibrosis (CF) population without a targeted therapy&#8230;. <\/p>\n<div class=\"clear\"><\/div>\n<p><a href=\"https:\/\/www.emilysentourage.org\/tr\/therapeutic-development-for-nonsense-mutations-the-final-frontier-of-cf\/\" class=\"excerpt-read-more\">Daha fazla bilgi edinin<\/a><\/p>","protected":false},"author":1704,"featured_media":11501,"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":"","_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":0,"_seopress_analysis_target_kw":"","_monsterinsights_skip_tracking":false,"_jetpack_newsletter_access":"","_jetpack_dont_email_post_to_subs":false,"_jetpack_newsletter_tier_id":0,"_jetpack_memberships_contains_paywalled_content":false,"_jetpack_hide_featured_image":false,"_jetpack_featured_image_duplicate_dismissed":0,"_jetpack_feature_clip_id":0,"_jetpack_memberships_contains_paid_content":false,"footnotes":"","jetpack_post_was_ever_published":false},"categories":[1],"tags":[],"class_list":["post-11486","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-uncategorized"],"acf":[],"jetpack_sharing_enabled":true,"jetpack_featured_media_url":"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/WistarInstitute.jpg","post_mailing_queue_ids":[],"_links":{"self":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/11486","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\/1704"}],"replies":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/comments?post=11486"}],"version-history":[{"count":5,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/11486\/revisions"}],"predecessor-version":[{"id":11522,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/11486\/revisions\/11522"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media\/11501"}],"wp:attachment":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media?parent=11486"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/categories?post=11486"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/tags?post=11486"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}