{"id":24319,"date":"2025-02-20T09:23:56","date_gmt":"2025-02-20T13:23:56","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?p=24319"},"modified":"2025-02-20T09:27:01","modified_gmt":"2025-02-20T13:27:01","slug":"accelerating-gene-therapy-delivery-for-cystic-fibrosis-emilys-entourage-to-convene-experts-at-2025-symposium","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/tr\/accelerating-gene-therapy-delivery-for-cystic-fibrosis-emilys-entourage-to-convene-experts-at-2025-symposium\/","title":{"rendered":"Kistik Fibrozis i\u00e7in Gen Terapisi Uygulamas\u0131n\u0131n H\u0131zland\u0131r\u0131lmas\u0131: Emily's Entourage 2025 Sempozyumunda Uzmanlar\u0131 Bir Araya Getirecek"},"content":{"rendered":"<p><strong>Philadelphia, PA-20 \u015eubat 2025<\/strong>-Kistik fibrozis (KF) toplulu\u011funun son 10%'si i\u00e7in ara\u015ft\u0131rmalar\u0131 h\u0131zland\u0131ran \u00f6nc\u00fc bir kar amac\u0131 g\u00fctmeyen kurulu\u015f olan Emily's Entourage (EE), 2025 Bilimsel Sempozyumuna ev sahipli\u011fi yapmaya haz\u0131rlan\u0131yor. <i>\"Genlerin \u0130letimi: KF Merce\u011finden Geli\u015fmeler ve Zorluklar,\"<\/i> 23-26 \u015eubat 2025 tarihleri aras\u0131nda Philadelphia, Pennsylvania'daki University City Science Center'daki Quorum'da ger\u00e7ekle\u015ftirilecektir.<\/p>\n<p><img fetchpriority=\"high\" decoding=\"async\" class=\"alignright wp-image-24324\" src=\"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/2025-Symposium-Graphic-1000x610.png\" alt=\"\" width=\"560\" height=\"372\" srcset=\"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/2025-Symposium-Graphic-300x200.png 300w, https:\/\/www.emilysentourage.org\/wp-content\/uploads\/2025-Symposium-Graphic-768x511.png 768w, https:\/\/www.emilysentourage.org\/wp-content\/uploads\/2025-Symposium-Graphic-18x12.png 18w, https:\/\/www.emilysentourage.org\/wp-content\/uploads\/2025-Symposium-Graphic-600x399.png 600w, https:\/\/www.emilysentourage.org\/wp-content\/uploads\/2025-Symposium-Graphic.png 1024w\" sizes=\"(max-width: 560px) 100vw, 560px\" \/>Bu y\u0131lki sempozyum, KF i\u00e7in n\u00fckleotid bazl\u0131 tedavilerin geli\u015ftirilmesindeki ilerlemenin s\u0131n\u0131rlar\u0131n\u0131 zorlamay\u0131 ve gen bazl\u0131 tedavilerin en kritik ve karma\u015f\u0131k zorluklar\u0131ndan birine odaklanmay\u0131 ama\u00e7lamaktad\u0131r: bunlar\u0131n iletimi. Her ne kadar ilerlemeler kaydedilmi\u015f olsa da, genetik materyalin etkili bir \u015fekilde iletilmesiyle ilgili engeller b\u00fcy\u00fck bir engel olmaya devam etmektedir. N\u00fckleotid bazl\u0131 tedaviler t\u00fcm KF toplulu\u011fu i\u00e7in umut vaat etse de, mevcut CFTR mod\u00fclat\u00f6r tedavilerinden faydalanamayan KF pop\u00fclasyonunun son 10%'sinde yer alan ki\u015filer i\u00e7in ihtiya\u00e7 \u00f6zellikle acildir. Bu grup i\u00e7in zaman \u00e7ok \u00f6nemlidir ve ilerlemenin geleneksel zaman \u00e7izelgelerinde ortaya \u00e7\u0131kmas\u0131n\u0131 beklemek i\u00e7in yer yoktur.<\/p>\n<p>\"D\u00fcnyan\u0131n en parlak ara\u015ft\u0131rmac\u0131lar\u0131n\u0131 ve d\u00fc\u015f\u00fcnce liderlerini bir odada bir araya getirmenin g\u00fcc\u00fcne inan\u0131yoruz. Amac\u0131m\u0131z a\u00e7\u0131k payla\u015f\u0131m\u0131 te\u015fvik etmek, i\u015fbirli\u011fini ate\u015flemek, b\u00fcy\u00fck d\u00fc\u015f\u00fcnmek ve ileriye d\u00f6n\u00fck yeni, h\u0131zland\u0131r\u0131lm\u0131\u015f yollar olu\u015fturmakt\u0131r,\" diyor MBE, EE kurucu orta\u011f\u0131 Emily Kramer-Golinkoff. \"KF bilim camias\u0131n\u0131n ve end\u00fcstrisinin i\u00e7inden ve d\u0131\u015f\u0131ndan \u00f6nde gelen beyinleri bir araya getiren bu sempozyum, \u00e7\u0131\u011f\u0131r a\u00e7an bu tedavilerin uygulanmas\u0131n\u0131 h\u0131zland\u0131rmaya haz\u0131rlan\u0131yor.\"<\/p>\n<p>Pennsylvania \u00dcniversitesi Perelman T\u0131p Fak\u00fcltesi'nde Isaac Ott Fizyoloji Profes\u00f6r\u00fc Kevin Foskett, PhD; Spirovant Sciences, Inc. CEO'su Joan Lau, PhD, MBA; ve Iowa \u00dcniversitesi'nde pediatri-pulmoner t\u0131p profes\u00f6r\u00fc Paul McCray, Jr, MD'nin ba\u015fkanl\u0131k edece\u011fi etkinlikte, hem KF alan\u0131nda hem de \u00f6tesinde akademi, biyoteknoloji ve ila\u00e7 sekt\u00f6r\u00fcnden 40'tan fazla d\u00fcnyaca \u00fcnl\u00fc ara\u015ft\u0131rmac\u0131, yenilik\u00e7i ve d\u00fc\u015f\u00fcnce lideri yer alacak. Bu uzmanlar, en son geli\u015fmeleri inceleyecek ve gen bazl\u0131 tedavinin t\u00fcm potansiyelini ortaya \u00e7\u0131karmada kritik bir ad\u0131m olan n\u00fckleotid bazl\u0131 tedavileri KF'li bireylere g\u00fcvenli ve etkili bir \u015fekilde sunman\u0131n benzersiz zorluklar\u0131na dalacaklar. \u00c7eviri f\u0131rsatlar\u0131na odaklanan bu sempozyum, sadece bilimi de\u011fil, ayn\u0131 zamanda KF tedavisinin manzaras\u0131n\u0131 d\u00f6n\u00fc\u015ft\u00fcrmek i\u00e7in i\u015fbirli\u011finin g\u00fcc\u00fcn\u00fc de ele al\u0131yor.<\/p>\n<p>2025 Sempozyumu, EE'nin nadir genetik mutasyonlar\u0131 nedeniyle mevcut CFTR mod\u00fclat\u00f6rlerinden yararlanamayan KF'li ki\u015filerin son 10%'si i\u00e7in terap\u00f6tik geli\u015fimi h\u0131zland\u0131rmaya y\u00f6nelik devam eden \u00e7abalar\u0131na dayanmaktad\u0131r. EE'nin \u00e7al\u0131\u015fmalar\u0131n\u0131n \u00f6nemli noktalar\u0131 \u015funlard\u0131r:<\/p>\n<ul>\n<li aria-level=\"1\">\u00d6d\u00fcllendirme <a href=\"https:\/\/www.emilysentourage.org\/tr\/awarded-grants\/\" target=\"_blank\" rel=\"noopener\">38 ara\u015ft\u0131rma hibesi<\/a> D\u00fcnya \u00e7ap\u0131ndaki en iyi ara\u015ft\u0131rmac\u0131lara $52,3 milyondan fazla devam fonu sa\u011flad\u0131.<\/li>\n<li aria-level=\"1\">Bir giri\u015fim filantropisi modelinin hayata ge\u00e7irilmesi <a href=\"https:\/\/www.emilysentourage.org\/tr\/emilys-entourage-celebrates-landmark-achievement-first-patient-dosed-with-novel-gene-therapy-developed-through-ees-visionary-model\/\" target=\"_blank\" rel=\"noopener\">\u015fu anda Faz 1\/2 klinik \u00e7al\u0131\u015fma y\u00fcr\u00fcten bir KF gen terapisi \u015firketi<\/a>.<\/li>\n<li aria-level=\"1\">Antibiyoti\u011fe diren\u00e7li enfeksiyonu olan 34'ten fazla ki\u015fiyi tedavi eden 37 faj geli\u015ftirildi.<\/li>\n<li aria-level=\"1\">Ba\u015flat\u0131l\u0131yor <a href=\"https:\/\/www.emilysentourage.org\/tr\/ctc\/\" target=\"_blank\" rel=\"noopener\">CF Clinical Trial Connect (CTC)<\/a>k\u00fcresel bir hasta veri taban\u0131 ve bu ultra nadir pop\u00fclasyon i\u00e7in i\u015fe al\u0131mlar\u0131 h\u0131zland\u0131rmak i\u00e7in Klinik Deneme E\u015fle\u015ftirme Program\u0131.<\/li>\n<\/ul>\n<p>EE Bilimsel Ba\u015f Sorumlusu Chandra Ghose, \"KF gen terapisi ara\u015ft\u0131rmalar\u0131nda devam eden ivmeden inan\u0131lmaz derecede gurur duyuyoruz, ancak ger\u00e7ekten bir fark yaratmak ve tedaviler geli\u015ftirmek i\u00e7in zorlu sorularla y\u00fczle\u015fmemiz ve disiplinler aras\u0131nda i\u015fbirli\u011fi yapmam\u0131z gerekti\u011finin fark\u0131nday\u0131z\" dedi. \"Bu kadar \u00e7e\u015fitli d\u00fc\u015f\u00fcnce liderlerini bir araya getirerek, yeni fikirleri ate\u015flemeyi, s\u0131n\u0131rlar\u0131 zorlamay\u0131 ve nihayetinde en \u00e7ok ihtiyac\u0131 olanlar i\u00e7in tedaviler geli\u015ftirme zaman \u00e7izelgesini h\u0131zland\u0131rmay\u0131 ama\u00e7l\u0131yoruz.\"<\/p>\n<p>EE, 2025 sempozyumu r\u00fcya tak\u0131m\u0131 sponsorlar\u0131na te\u015fekk\u00fcr eder, <b>Viatris,<\/b> <b>4D Molek\u00fcler Terap\u00f6tikler<\/b>, <b>Vertex Pharmaceuticals, Inc, <\/b>mor d\u00fczey sponsorlar, <b>ReCode Therapeutics ve Boomer Esiason Vakf\u0131,<\/b> ve daha fazlas\u0131 bu kritik etkinli\u011fi ve sa\u011flad\u0131\u011f\u0131 ilerlemeyi m\u00fcmk\u00fcn k\u0131lmaktad\u0131r.<\/p>\n<p>&nbsp;<\/p>\n<p><b>Emily'nin \u00c7evresi Hakk\u0131nda<\/b><\/p>\n<p>Emily's Entourage, mevcut mutasyon hedefli tedavilerden yararlanamayan 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, \u015fu anda klinik a\u015famada olan bir KF gen terapisi \u015firketi kurmu\u015f, klinik deneylere kat\u0131l\u0131m\u0131 h\u0131zland\u0131rmak i\u00e7in bir hasta veri taban\u0131 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<a href=\"https:\/\/www.emilysentourage.org\/tr\/\"> emilysentourage.org<\/a>.<\/p>\n<p>&nbsp;<\/p>\n<p><b>Medya \u0130leti\u015fim<\/b><\/p>\n<p>Hannah Kleckner<\/p>\n<p>Pazarlama ve \u0130leti\u015fim Direkt\u00f6r\u00fc<\/p>\n<p><a href=\"mailto:hannah@emilysentourage.org\">hannah@emilysentourage.org<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Philadelphia, PA-20 \u015eubat 2025-Kistik fibrozis (KF) toplulu\u011funun son 10%\u201csi i\u00e7in ara\u015ft\u0131rmalar\u0131 h\u0131zland\u0131ran \u00f6nc\u00fc bir kar amac\u0131 g\u00fctmeyen kurulu\u015f olan Emily's Entourage (EE), \u201dGenlerin Da\u011f\u0131t\u0131m\u0131\" ba\u015fl\u0131kl\u0131 2025 Bilimsel Sempozyumuna ev sahipli\u011fi yapmaya haz\u0131rlan\u0131yor: KF Merce\u011finden \u0130lerlemeler ve Zorluklar\" ba\u015fl\u0131kl\u0131 2025 Bilimsel Sempozyumuna 23-26 \u015eubat 2025 tarihleri aras\u0131nda University City Bilim Merkezindeki Quorum'da ev sahipli\u011fi yapacak... <\/p>\n<div class=\"clear\"><\/div>\n<p><a href=\"https:\/\/www.emilysentourage.org\/tr\/accelerating-gene-therapy-delivery-for-cystic-fibrosis-emilys-entourage-to-convene-experts-at-2025-symposium\/\" class=\"excerpt-read-more\">Daha fazla bilgi edinin<\/a><\/p>","protected":false},"author":1719,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_seopress_robots_primary_cat":"none","_seopress_titles_title":"","_seopress_titles_desc":"","_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":[203,204,188,111,183],"class_list":["post-24319","post","type-post","status-publish","format-standard","hentry","category-press-releases","tag-2025-scientific-symposium","tag-ee-research","tag-gene-therapy","tag-research","tag-symposium"],"acf":[],"jetpack_featured_media_url":"","jetpack_sharing_enabled":true,"post_mailing_queue_ids":[],"_links":{"self":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/24319","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\/1719"}],"replies":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/comments?post=24319"}],"version-history":[{"count":5,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/24319\/revisions"}],"predecessor-version":[{"id":24328,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/24319\/revisions\/24328"}],"wp:attachment":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media?parent=24319"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/categories?post=24319"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/tags?post=24319"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}