{"id":27109,"date":"2026-09-08T14:12:48","date_gmt":"2026-09-08T18:12:48","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?p=27109"},"modified":"2026-09-08T14:12:48","modified_gmt":"2026-09-08T18:12:48","slug":"emilys-entourage-awards-2026-translational-research-grant-to-advance-novel-gene-editing-delivery-for-cystic-fibrosis","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/tr\/emilys-entourage-awards-2026-translational-research-grant-to-advance-novel-gene-editing-delivery-for-cystic-fibrosis\/","title":{"rendered":"Emily\u2019s Entourage, Kistik Fibrozis i\u00e7in Yeni Nesil Gen D\u00fczenleme Da\u011f\u0131t\u0131m\u0131n\u0131 \u0130lerletmek Amac\u0131yla 2026 \u00c7eviri Ara\u015ft\u0131rmalar\u0131 Hibesini Veriyor"},"content":{"rendered":"

Yeni lipid nanopartik\u00fcl platformu, kistik fibrozis i\u00e7in kal\u0131c\u0131 bir tedaviye yakla\u015f\u0131lmas\u0131n\u0131 sa\u011flayarak, CFTR mutasyonlar\u0131n\u0131 d\u00fczeltmek \u00fczere gen d\u00fczenleyicileri g\u00fcvenli ve verimli bir \u015fekilde ula\u015ft\u0131rmay\u0131 ama\u00e7lamaktad\u0131r.<\/span><\/i><\/p>\n

\"\"

Emily's Entourage (EE), Case Western Reserve University'de Biyomedikal M\u00fchendisli\u011fi M. Frank Rudy ve Margaret Domiter Rudy Profes\u00f6r\u00fc olan Do\u00e7. Dr. Zheng-Rong Lu'ya 2026 y\u0131l\u0131ndaki be\u015finci D\u00f6n\u00fc\u015ft\u00fcr\u00fcc\u00fc Ara\u015ft\u0131rma Hibesini vermi\u015ftir.<\/em><\/p><\/div>\n

Lower Merion, PA\u20148 Eyl\u00fcl 2026\u2014<\/span>Mevcut mutasyon odakl\u0131 tedavilerden yararlanamayan kistik fibrozis (KF) hastalar\u0131 i\u00e7in ara\u015ft\u0131rmalar\u0131 h\u0131zland\u0131ran yenilik\u00e7i bir 501(c)(3) kurulu\u015fu olan Emily's Entourage (EE), Case Western Reserve \u00dcniversitesi Biyomedikal M\u00fchendisli\u011fi M. Frank Rudy ve Margaret Domiter Rudy Profes\u00f6r\u00fc Do\u00e7. Dr. Zheng-Rong Lu'ya verilen 2026 y\u0131l\u0131ndaki be\u015finci \u00c7eviri Ara\u015ft\u0131rmas\u0131 Hibe Deste\u011fini duyurmaktan gurur duyar.<\/span><\/p>\n

Dr. Lu\u2019nun projesi, gen d\u00fczenleme tedavileri i\u00e7in yeni nesil bir uygulama platformu geli\u015ftmeyi ama\u00e7lamaktad\u0131r. Bu proje, bu alandaki en \u00f6nemli engellerden birini ortadan kald\u0131rmay\u0131 ve son a\u015famadaki 10% hastalar\u0131 da dahil olmak \u00fczere kistik fibroz hastalar\u0131 i\u00e7in kal\u0131c\u0131 bir tedavi yakla\u015f\u0131m\u0131 olarak gen d\u00fczenlemenin potansiyelini ortaya \u00e7\u0131karmaya yard\u0131mc\u0131 olmay\u0131 hedeflemektedir.<\/span><\/p>\n

Gen d\u00fczenleme, altta yatan genetik mutasyonu d\u00fczeltme ve normal CFTR i\u015flevini geri kazand\u0131rma potansiyeli sayesinde, uzun s\u00fcredir kistik fibroz (CF) tedavisinde d\u00f6n\u00fc\u015ft\u00fcr\u00fcc\u00fc bir yakla\u015f\u0131m olarak umut vaat etmektedir. Ancak, gen d\u00fczenleyicilerin akci\u011ferlerdeki uygun h\u00fccrelere g\u00fcvenli ve verimli bir \u015fekilde ula\u015ft\u0131r\u0131lmas\u0131, bu alandaki ilerlemeyi s\u0131n\u0131rlayan zorluklar yaratm\u0131\u015ft\u0131r. Hastal\u0131\u011f\u0131n temel nedenini ortadan kald\u0131rma ve t\u00fcm CF mutasyonlar\u0131 i\u00e7in kal\u0131c\u0131 terap\u00f6tik fayda sa\u011flama potansiyeli g\u00f6z \u00f6n\u00fcne al\u0131nd\u0131\u011f\u0131nda, bu zorlu\u011fun a\u015f\u0131lmas\u0131 \u00f6zellikle 10% i\u00e7in b\u00fcy\u00fck \u00f6nem ta\u015f\u0131maktad\u0131r.<\/span><\/p>\n

Dr. Lu'nun ara\u015ft\u0131rmas\u0131, gen d\u00fczenleme ara\u00e7lar\u0131n\u0131 do\u011frudan h\u00fccrelerin i\u00e7ine ta\u015f\u0131mak i\u00e7in tasarlanm\u0131\u015f m\u00fchendislik \u00fcr\u00fcn\u00fc ta\u015f\u0131y\u0131c\u0131lar olan \u00e7ok fonksiyonlu iyonla\u015ft\u0131r\u0131labilir lipid nanopartik\u00fcllerin (LNP'ler) geli\u015ftirilmesi yoluyla bu engeli a\u015fmaya odaklanmaktad\u0131r. LNP'ler mRNA a\u015f\u0131lar\u0131 gibi uygulamalarda halihaz\u0131rda g\u00fcvenli bir \u015fekilde kullan\u0131lsa da mevcut form\u00fclasyonlar, kistik fibrozis (CF) gibi hastal\u0131klar i\u00e7in protein bazl\u0131 gen d\u00fczenleyicileri iletmek \u00fczere optimize edilmemi\u015ftir.<\/span><\/p>\n

Bu proje, \u00f6zellikle bu s\u0131n\u0131rlamalar\u0131 ele almak i\u00e7in tasarlanm\u0131\u015f yeni bir biyobozunur LNP s\u0131n\u0131f\u0131 sunmaktad\u0131r. DNA veya mRNA tabanl\u0131 yakla\u015f\u0131mlar\u0131n aksine, bu platform gen d\u00fczenleme makinelerini, yabanc\u0131 genetik materyalin ekspresyonunu gerektirmeyen ve uzun s\u00fcreli veya kontrols\u00fcz gen aktivitesiyle ili\u015fkili g\u00fcvenlik endi\u015felerini azaltabilecek ribon\u00fckleoproteinler (RNP'ler) \u015feklinde sunar.<\/span><\/p>\n

Yeni geli\u015ftirilen nanopartik\u00fcller sulu ortamlarda kararl\u0131 olacak, sert organik \u00e7\u00f6z\u00fcc\u00fclerin kullan\u0131m\u0131ndan ka\u00e7\u0131nacak ve v\u00fccutta biyouyumlu bile\u015fenlere ayr\u0131\u015facak \u015fekilde tasarlanm\u0131\u015ft\u0131r. Erken d\u00f6nem \u00e7al\u0131\u015fmalar, bu LNP'lerin daha d\u00fc\u015f\u00fck dozlarda daha verimli h\u00fccre i\u00e7i da\u011f\u0131l\u0131m ve gen d\u00fczenlemesi sa\u011flayabildi\u011fini, ayn\u0131 zamanda \u00f6nceki sistemlere k\u0131yasla geli\u015fmi\u015f g\u00fcvenlik profilleri sergiledi\u011fini g\u00f6stermektedir.<\/span><\/p>\n

Bu platformu kullanan Dr. Lu\u2019nun ekibi, Case Western Reserve \u00dcniversitesi'nde mevcut olan klinik \u00f6ncesi modellerde, \u00f6ncelikli olarak W1282X mutasyonuna odaklanarak CFTR mutasyonlar\u0131n\u0131 hedefleyen gen d\u00fczenleyicilerin iletimini optimize edecektir. Proje, daha ileritranslational (d\u00f6n\u00fc\u015ft\u00fcr\u00fcc\u00fc) geli\u015ftirme i\u00e7in \u00f6nc\u00fc bir terap\u00f6tik aday belirleme amac\u0131yla hem gen d\u00fczenleme verimlili\u011fini hem de CFTR fonksiyonunun restorasyonunu de\u011ferlendirecektir. Gen d\u00fczenleyicilerin daha verimli iletimini sa\u011flayarak bu yakla\u015f\u0131m, Kistik Fibrozis'i (CF) kayna\u011f\u0131nda d\u00fczelten tedavilere alan\u0131 daha da yakla\u015ft\u0131rabilir.<\/span><\/p>\n

\u201cGen d\u00fczenleme, K\u0130'yi tedavi etme \u015feklimizi k\u00f6kten de\u011fi\u015ftirme potansiyeline sahiptir, ancak yaln\u0131zca bu tedavileri g\u00fcvenli ve etkili bir \u015fekilde ula\u015ft\u0131rabilirsek,\u201d dedi Dr. Lu. \u201cBu \u00e7al\u0131\u015fma, \u00f6zellikle \u015fu anda hi\u00e7bir tedavi se\u00e7ene\u011fi olmayan K\u0130 hastalar\u0131 i\u00e7in bunu m\u00fcmk\u00fcn k\u0131labilecek bir da\u011f\u0131t\u0131m sistemi geli\u015ftirmeye odaklanm\u0131\u015ft\u0131r.\u201d<\/span><\/p><\/blockquote>\n

Kistik fibroz (CF) hastalar\u0131n\u0131n yakla\u015f\u0131k %\u2019si, mevcut CFTR mod\u00fclat\u00f6rleri i\u00e7in uygun olmayan genetik mutasyonlara sahiptir. EE, bu tedavilerden fayda g\u00f6rmeyen t\u00fcm bireyler i\u00e7in ara\u015ft\u0131rma ve tedavi geli\u015ftirme \u00e7al\u0131\u015fmalar\u0131n\u0131 h\u0131zland\u0131rmaya ve hi\u00e7 kimsenin geride kalmamas\u0131n\u0131 sa\u011flamaya kararl\u0131d\u0131r.<\/span><\/p>\n

\u201cEE\u2019nin Bilimsel Direkt\u00f6r\u00fc Dr. Chandra Ghose, \u201dBu proje, bu alandaki en kritik zorluklardan biri olan \u201cuygulama\u201d konusunu ele al\u0131yor,\u201d dedi. \u201cB\u00f6ylelikle, bu proje, sadece teoride de\u011fil, pratikte de, hastal\u0131\u011f\u0131n temel nedenini ortadan kald\u0131ran bir tedavi bekleyen son 10% grubundaki hastalar da dahil olmak \u00fczere, kistik fibroz hastalar\u0131 i\u00e7in gen d\u00fczenlemeyi ger\u00e7ek bir tedavi yolu haline getirmeye yard\u0131mc\u0131 olma potansiyeline sahiptir.\u201d<\/span><\/p>\n

 <\/p><\/blockquote>\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 f\u0131rsatlar\u0131 hakk\u0131nda daha fazla bilgi edinmek i\u00e7in \u015fu adresi ziyaret edin <\/span>https:\/\/www.emilysentourage.org\/funding-opportunities\/<\/span><\/a>.<\/span><\/p>\n

 <\/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

 <\/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":"

Novel lipid nanoparticle platform aims to safely and efficiently deliver gene editors to correct CFTR mutations, bringing the field closer to a durable treatment for cystic fibrosis Lower Merion, PA\u2014September 8, 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… <\/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":"Emily\u2019s Entourage Awards 2026 Translational Research Grant to Advance Novel Gene Editing Delivery for Cystic Fibrosis","_seopress_titles_desc":"Emily\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 a 2026 Translational Research Grant awarded to Zheng-Rong Lu, PhD, M. Frank Rudy and Margaret Domiter Rudy Professor of Biomedical Engineering at Case Western Reserve University.","_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":[223,204,271,270,226],"class_list":["post-27109","post","type-post","status-publish","format-standard","hentry","category-press-releases","tag-ee-grant","tag-ee-research","tag-gene-editing","tag-new-cf-research","tag-translational-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\/27109","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=27109"}],"version-history":[{"count":1,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/27109\/revisions"}],"predecessor-version":[{"id":27112,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/27109\/revisions\/27112"}],"wp:attachment":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media?parent=27109"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/categories?post=27109"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/tags?post=27109"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}