{"id":27038,"date":"2026-08-18T09:00:49","date_gmt":"2026-08-18T13:00:49","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?p=27038"},"modified":"2026-08-18T12:34:27","modified_gmt":"2026-08-18T16:34:27","slug":"new-2026-emilys-entourage-translational-research-grant-to-develop-mucus-penetrating-gene-editing-delivery-for-cystic-fibrosis","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/tr\/new-2026-emilys-entourage-translational-research-grant-to-develop-mucus-penetrating-gene-editing-delivery-for-cystic-fibrosis\/","title":{"rendered":"Kistik Fibrozis i\u00e7in Mukusa N\u00fcfuz Eden Gen D\u00fczenleme Da\u011f\u0131t\u0131m\u0131 Geli\u015ftirmek Amac\u0131yla Yeni 2026 Emily\u2019s Entourage \u00c7eviri Ara\u015ft\u0131rmas\u0131 Hibesi"},"content":{"rendered":"

Kistik fibroz mukus bariyerini a\u015fmak i\u00e7in tasarlanm\u0131\u015f m\u00fchendislik \u00fcr\u00fcn\u00fc lipit nanopartik\u00fcller, inhalasyon yoluyla hedeflenmi\u015f gen d\u00fczenlemesini sa\u011flayabilir<\/span><\/i><\/p>\n

\"\"

Emily\u2019s Entourage (EE), Johns Hopkins \u00dcniversitesi Malzeme Bilimi ve M\u00fchendisli\u011fi Departman\u0131 ile Pediatri Departman\u0131nda Ara\u015ft\u0131rma Yard\u0131mc\u0131 Profes\u00f6r\u00fc olan Do\u00e7. Dr. Xizhen Lian\u2019a 2026 y\u0131l\u0131ndaki ikinci \u00c7eviri Ara\u015ft\u0131rma Hibe'sini vermi\u015ftir.<\/em><\/p><\/div>\n

Lower Merion, PA\u201418 A\u011fustos 2026\u2014Mevcut mutasyon odakl\u0131 tedavilerden yararlanmayan kistik fibrozis (KF) hastalar\u0131 i\u00e7in ara\u015ft\u0131rmalar\u0131 h\u0131zland\u0131ran yenilik\u00e7i bir 501(c)(3) kurulu\u015fu olan Emily\u2019s Entourage (EE), Johns Hopkins \u00dcniversitesi Malzeme Bilimi ve M\u00fchendisli\u011fi Departman\u0131 ile Pediatri Departman\u0131'nda Ara\u015ft\u0131rma Yard\u0131mc\u0131 Profes\u00f6r\u00fc olan Dr. Xizhen Lian'a verilen 2026 y\u0131l\u0131ndaki ikinci \u00c7eviri Ara\u015ft\u0131rmas\u0131 Hibesini duyurmaktan gurur duyar.<\/span><\/p>\n

Ortak ara\u015ft\u0131rmac\u0131 Dr. Justin Hanes ile birlikte Dr. Lian'\u0131n projesi, Kistik Fibrozis (KF) hava yollar\u0131ndaki kal\u0131n mukusa n\u00fcfuz edebilen\u2014hastal\u0131\u011f\u0131 kayna\u011f\u0131nda tedavi etmenin \u00f6n\u00fcndeki en b\u00fcy\u00fck engellerden biri\u2014gen d\u00fczenleme tedavileri i\u00e7in yeni bir solunabilir da\u011f\u0131t\u0131m platformu geli\u015ftirmeyi ama\u00e7lamaktad\u0131r.<\/span><\/p>\n

Gen d\u00fczenleme, mevcut CFTR mod\u00fclat\u00f6rlerinden fayda g\u00f6rmeyen bireylerde g\u00f6r\u00fclen son 10%'yi de i\u00e7eren, CF'ye neden olan altta yatan genetik mutasyonlar\u0131 d\u00fczeltme konusunda b\u00fcy\u00fck umut vaat etmektedir. Ancak, bu alandaki en b\u00fcy\u00fck zorluklardan biri, yo\u011fun ve yap\u0131\u015fkan mukusun tedavilerin hedeflerine ula\u015fmas\u0131n\u0131 engelleyebilece\u011fi akci\u011ferlerdeki do\u011fru h\u00fccrelere gen d\u00fczenleyicileri g\u00fcvenli ve etkili bir \u015fekilde ula\u015ft\u0131rmakt\u0131r.<\/span><\/p>\n

Dr. Lian ve Dr. Hanes, gen d\u00fczenleme ara\u00e7lar\u0131n\u0131 inhalasyonu takiben do\u011frudan havayolu h\u00fccrelerine ta\u015f\u0131mak i\u00e7in tasarlanm\u0131\u015f minik ta\u015f\u0131ma ara\u00e7lar\u0131 olan mukusa n\u00fcfuz eden lipit nanopartik\u00fcller (LNP'ler) tasarlayarak bu zorlu\u011fun \u00fcstesinden gelmeyi ama\u00e7lamaktad\u0131r.<\/span><\/p>\n

\u00d6nceki LNP tabanl\u0131 yakla\u015f\u0131mlar sistemik olarak verildi\u011finde umut vadetti\u011fini g\u00f6sterse de, hedeflenmeyen organlardaki da\u011f\u0131l\u0131m ve toksisite ile ilgili zorluklar ortaya \u00e7\u0131karabilirler. Buna kar\u015f\u0131l\u0131k, bu proje akci\u011ferlere lokal uygulamaya odaklanmaktad\u0131r; bu yakla\u015f\u0131m hassasiyeti art\u0131rmay\u0131, hedeflenmeyen etkileri azaltmay\u0131 ve gelecekteki bir tedavinin K\u0130 hastalar\u0131nda nas\u0131l uygulanabilece\u011fini daha iyi yans\u0131tmay\u0131 ama\u00e7lamaktad\u0131r.<\/span><\/p>\n

Ekibin stratejisi, mukusa n\u00fcfuz eden par\u00e7ac\u0131k teknolojisindeki \u00f6nceki ilerlemelere dayanmakta ve y\u00fczey Polietilen glikol (PEG) kaplamalar\u0131n\u0131 optimize ederek bunlar\u0131 yeni nesil LNP'lere uygulamakta; b\u00f6ylece nanopartik\u00fcllerin hapsolmak yerine KF balgam\u0131 i\u00e7inde hareket etmesine olanak tan\u0131maktad\u0131r.<\/span><\/p>\n

LNP'lerin mukus boyunca dif\u00fczyon yetene\u011fini ve hava yolu h\u00fccrelerine ula\u015fma kapasitesini art\u0131ran bu platform, hava yolu epiteli genelinde geni\u015f tabanl\u0131 ve d\u00fc\u015f\u00fck d\u00fczeyli gen d\u00fczenlemesine olanak tan\u0131mak \u00fczere tasarlanm\u0131\u015ft\u0131r; bu yakla\u015f\u0131m, k\u0131sm\u00ee bir d\u00fczeltmenin bile anlaml\u0131 akci\u011fer fonksiyonunu eski haline getirmek i\u00e7in yeterli olabilece\u011fini \u00f6ne s\u00fcren \u00f6nceki ara\u015ft\u0131rmalarla desteklenmektedir.<\/span><\/p>\n

Proje, KF akci\u011ferlerindeki ko\u015fullar\u0131 daha yak\u0131ndan yans\u0131tan, hastadan elde edilen havayolu h\u00fccre modellerinde ve mukus a\u015f\u0131r\u0131 salg\u0131layan hayvan modellerinde tasarlanm\u0131\u015f LNP'leri de\u011ferlendirecektir. Havayollar\u0131nda ila\u00e7 sal\u0131n\u0131m\u0131n\u0131 ilerletmenin yan\u0131 s\u0131ra bu \u00e7al\u0131\u015fma, mevcut akci\u011fer hedefli sal\u0131n\u0131m stratejileriyle etkili bir \u015fekilde ula\u015f\u0131lamayan gastrointestinal sistem gibi di\u011fer etkilenen mukoza dokular\u0131na gen d\u00fczenleme yakla\u015f\u0131mlar\u0131n\u0131n geni\u015fletilmesine yard\u0131mc\u0131 olabilir.<\/span><\/p>\n

\u201cMukus bariyerini a\u015fmak, KF i\u00e7in etkili genetik tedaviler geli\u015ftirmenin en kritik zorluklar\u0131ndan biridir,\u201d dedi Dr. Lian. \u201cBu bariyerden ge\u00e7ip do\u011fru h\u00fccrelere ula\u015fabilen nanopartik\u00fcller tasarlayarak, halihaz\u0131rda CFTR mod\u00fclat\u00f6r tedavilerinden yararlanamayan KF hastalar\u0131na fayda sa\u011flayabilecek gen d\u00fczenleme yakla\u015f\u0131mlar\u0131n\u0131 m\u00fcmk\u00fcn k\u0131lmay\u0131 ama\u00e7l\u0131yoruz.\u201d<\/span><\/p><\/blockquote>\n

\u201cEmily\u2019s Entourage\u2019\u0131n Bilimsel Direkt\u00f6r\u00fc Dr. Chandra Ghose, \u201dBu proje, bu alandaki ilerlemeyi s\u0131n\u0131rlayan temel bir engeli ele al\u0131yor: CF mukusu arac\u0131l\u0131\u011f\u0131yla tedavilerin etkili bir \u015fekilde ula\u015ft\u0131r\u0131lamamas\u0131,\u201c dedi. \u201dGen d\u00fczenleyicilerin do\u011frudan solunum yollar\u0131na hedefli bir \u015fekilde ula\u015ft\u0131r\u0131lmas\u0131n\u0131 sa\u011flayarak, bu \u00e7al\u0131\u015fma 10% i\u00e7in daha g\u00fcvenli ve daha hassas tedavi stratejilerinin geli\u015ftirilmesine katk\u0131da bulunma potansiyeline sahip ve bizi hastal\u0131\u011f\u0131n temel nedenini ele alan tedavilere bir ad\u0131m daha yakla\u015ft\u0131r\u0131yor.\u201d<\/span><\/p>\n

Emily's Entourage'\u0131n (EE) Hibe Program\u0131 Hakk\u0131nda<\/b><\/p>\n

Emily's Entourage's (EE) Hibrit Program\u0131, mevcut mutasyon odakl\u0131 tedavilerden yararlanamayan kistik fibrozisli (KF) hastalar i\u00e7in ara\u015ft\u0131rmalar\u0131 ve terap\u00f6tik geli\u015ftirmeyi h\u0131zland\u0131rmak amac\u0131yla finansman sa\u011flamaktad\u0131r. EE; \u00e7eviri hibeleri, i\u015f birli\u011fi hibeleri ve klinik \u00f6ncesi ke\u015fif hibelerinin yan\u0131 s\u0131ra risk sermayesi hay\u0131rseverli\u011fi yat\u0131r\u0131mlar\u0131 dahil olmak \u00fczere \u00e7e\u015fitli finansman mekanizmalar\u0131n\u0131 desteklemektedir.<\/span><\/p>\n

Bug\u00fcne kadar EE, d\u00fcnyan\u0131n d\u00f6rt bir yan\u0131ndan \u00e7ok disiplinli ekibe milyonlarca dolar hibe etmi\u015f ve takip eden fonlarda milyonlarca dolar\u0131n daha g\u00fcvence alt\u0131na al\u0131nmas\u0131na yard\u0131mc\u0131 olmu\u015ftur. Verilen hibeleri incelemek i\u00e7in ziyaret edin <\/span>https:\/\/www.emilysentourage.org\/awarded-grants\/<\/span><\/a>. EE'nin finansman\u0131 hakk\u0131nda daha fazla bilgi edinmek i\u00e7in f\u0131rsatlar\u0131 ziyaret edin<\/span>https:\/\/www.emilysentourage.org\/funding-opportunities\/<\/span><\/a>.<\/span><\/p>\n

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

Emily\u2019s Entourage (EE), mevcut mutasyon hedefli tedavilerden fayda g\u00f6remeyen kistik fibroz (CF) hastalar\u0131n\u0131n son a\u015famas\u0131ndaki bireyler i\u00e7in yeni tedaviler ve bir \u00e7are bulunmas\u0131na y\u00f6nelik ara\u015ft\u0131rmalar\u0131 h\u0131zland\u0131ran yenilik\u00e7i bir 501(c)3 kurulu\u015fudur. 2011 y\u0131l\u0131ndan bu yana EE, milyonlarca dolarl\u0131k ara\u015ft\u0131rma hibesi vermi\u015f, klinik a\u015famadaki bir CF gen terapisi \u015firketi kurmu\u015f, k\u00fcresel klinik deneme kat\u0131l\u0131m\u0131n\u0131 h\u0131zland\u0131rmak i\u00e7in Clinical Trial Connect (CTC) hasta veritaban\u0131n\u0131 ve klinik deneme e\u015fle\u015ftirme program\u0131n\u0131 geli\u015ftirmi\u015f ve y\u00fcksek etkili ara\u015ft\u0131rma ve ila\u00e7 geli\u015ftirme \u00e7al\u0131\u015fmalar\u0131n\u0131 te\u015fvik etmek i\u00e7in d\u00fcnya \u00e7ap\u0131nda \u00e7aba sarf 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>.<\/span><\/p>\n

Medya \u0130leti\u015fim<\/b><\/p>\n

Hannah Kleckner<\/span><\/p>\n

Pazarlama ve \u0130leti\u015fim Direkt\u00f6r\u00fc<\/span><\/p>\n

hannah@emilysentourage.org<\/span><\/p>\n

 <\/p>","protected":false},"excerpt":{"rendered":"

Engineered lipid nanoparticles designed to overcome the cystic fibrosis mucus barrier could enable targeted gene editing through inhalation Lower Merion, PA\u2014August 18, 2026\u2014Emily\u2019s Entourage (EE), an innovative 501(c)(3) organization accelerating research for individuals with cystic fibrosis (CF) who do not benefit from current mutation-targeted therapies, is proud to announce its second 2026 Translational Research Grant… <\/p>\n

<\/div>\n

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_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_feature_clip_id":0,"_jetpack_memberships_contains_paid_content":false,"footnotes":"","jetpack_post_was_ever_published":false},"categories":[178],"tags":[197,223,204,271,270,218],"class_list":["post-27038","post","type-post","status-publish","format-standard","hentry","category-press-releases","tag-cf-research","tag-ee-grant","tag-ee-research","tag-gene-editing","tag-new-cf-research","tag-research-grant"],"acf":[],"jetpack_sharing_enabled":true,"jetpack_featured_media_url":"","post_mailing_queue_ids":[],"_links":{"self":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/27038","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=27038"}],"version-history":[{"count":4,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/27038\/revisions"}],"predecessor-version":[{"id":27043,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/27038\/revisions\/27043"}],"wp:attachment":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media?parent=27038"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/categories?post=27038"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/tags?post=27038"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}