{"id":21076,"date":"2024-05-29T09:00:06","date_gmt":"2024-05-29T13:00:06","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?p=21076"},"modified":"2024-05-29T08:53:09","modified_gmt":"2024-05-29T12:53:09","slug":"emilys-entourage-awards-858932-in-inaugural-collaborative-research-grant-for-novel-approach-to-targeted-airway-delivery-in-cystic-fibrosis-treatments","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/tr\/emilys-entourage-awards-858932-in-inaugural-collaborative-research-grant-for-novel-approach-to-targeted-airway-delivery-in-cystic-fibrosis-treatments\/","title":{"rendered":"Emily's Entourage, Kistik Fibrozis Tedavilerinde Hedefe Y\u00f6nelik Hava Yolu Da\u011f\u0131t\u0131m\u0131na Yeni Yakla\u015f\u0131m i\u00e7in $858,932 A\u00e7\u0131l\u0131\u015f \u0130\u015fbirli\u011fi Ara\u015ft\u0131rma Bursu Kazand\u0131"},"content":{"rendered":"

EE'den yeni sa\u011flanan fon sayesinde Iowa \u00dcniversitesi, CHOP ve Rochester \u00dcniversitesi'nden ara\u015ft\u0131rma ekipleri, geli\u015fmi\u015f yeni adeno-ili\u015fkili vir\u00fcs (AAV) kapsid varyantlar\u0131ndan yararlanarak \u015fu anda mevcut olan kistik fibroz tedavilerindeki mevcut s\u0131n\u0131rlamalar\u0131n \u00fcstesinden gelmeyi ama\u00e7lamaktad\u0131r.<\/i><\/p>\n

\"From

Yukar\u0131dan A\u015fa\u011f\u0131ya: Paul McCray, Jr, MD; Beverly Davidson, PhD; John Lueck, PhD<\/p><\/div>\n

Lower Merion, PA - 29 May\u0131s 2024<\/strong> - Mevcut CFTR mod\u00fclat\u00f6rlerinden yararlanamayan kistik fibrozisli (KF) ki\u015filerin son 10%'si i\u00e7in yeni tedaviler ve bir tedavi ara\u015ft\u0131rmas\u0131n\u0131 h\u0131zland\u0131ran yenilik\u00e7i bir 501(c)3 olan Emily's Entourage (EE), bug\u00fcn $858,932 Ortak Hibe <\/a>Paul McCray, Jr, MD, Iowa \u00dcniversitesi'nde pediatri-pulmoner t\u0131p profes\u00f6r\u00fc; Beverly Davidson, PhD, Raymond G. Perelman H\u00fccresel ve Molek\u00fcler Terap\u00f6tikler Merkezi direkt\u00f6r\u00fc ve Philadelphia \u00c7ocuk Hastanesi (CHOP) ba\u015f bilimsel strateji sorumlusu, Pennsylvania \u00dcniversitesi Perelman T\u0131p Fak\u00fcltesi'nde patoloji ve laboratuvar t\u0131bb\u0131 profes\u00f6r\u00fc; ve John Lueck, PhD, Rochester \u00dcniversitesi T\u0131p ve Di\u015f Hekimli\u011fi Fak\u00fcltesi'nde farmakoloji ve fizyoloji do\u00e7enti. \u00d6d\u00fcl, T\u00fcrkiye'deki ilk EE'nin \u0130\u015fbirli\u011fi Hibesi <\/a>program\u0131, yeni KF terap\u00f6tiklerinin geli\u015ftirilmesindeki zorluklar\u0131n ortakla\u015fa \u00fcstesinden gelmek i\u00e7in farkl\u0131 uzmanl\u0131k alanlar\u0131na sahip birden fazla ara\u015ft\u0131rmac\u0131y\u0131 bir araya getirerek \u00e7\u0131\u011f\u0131r a\u00e7an KF tedavi ara\u015ft\u0131rmalar\u0131n\u0131 h\u0131zland\u0131rmak i\u00e7in yeni ba\u015flat\u0131lan bir finansman giri\u015fimidir.<\/p>\n

KF, CFTR genindeki mutasyonlar\u0131n neden oldu\u011fu ve akci\u011ferleri, sindirim sistemini ve di\u011fer organ sistemlerini etkileyen \u00f6l\u00fcmc\u00fcl bir genetik hastal\u0131kt\u0131r. CFTR mod\u00fclat\u00f6r tedavilerindeki \u00f6nemli geli\u015fmelere ra\u011fmen, KF'li bireylerin yakla\u015f\u0131k 10%'si spesifik mutasyonlar, intolerans veya mevcut ila\u00e7lara suboptimal yan\u0131t nedeniyle hala etkili tedavi se\u00e7eneklerinden yoksundur.<\/p>\n

Dr. Paul McCray liderli\u011finde Beverly Davidson, PhD ve John Lueck, PhD ile birlikte finanse edilen \"Yeni Adeno-ili\u015fkili Vir\u00fcs (AAV) Kapsid Varyantlar\u0131 ile Geli\u015ftirilmi\u015f Hava Yolu \u0130letimi ve Terap\u00f6tik Kargo Da\u011f\u0131t\u0131m\u0131\" projesi, geli\u015ftirilmi\u015f terap\u00f6tik kargo da\u011f\u0131t\u0131m\u0131 yoluyla etkili tedavilerin \u00f6n\u00fcndeki bilinen engelleri a\u015farak yeni KF terap\u00f6tik geli\u015fimini ilerletmeyi ama\u00e7lamaktad\u0131r.<\/p>\n

Ara\u015ft\u0131rma ekibi, KF'li ki\u015filerde CFTR i\u015flevini geri kazand\u0131ran geli\u015fmekte olan teknolojilerin da\u011f\u0131t\u0131m\u0131n\u0131 geli\u015ftirmeyi ama\u00e7lamaktad\u0131r. Bu yakla\u015f\u0131m, zay\u0131f transd\u00fcksiyon verimlili\u011fi gibi zorluklar\u0131 ele almak i\u00e7in AAV kapsid varyantlar\u0131n\u0131 kullanarak, \u015fu anda etkili tedavilerden yoksun olan KF'li 10% i\u00e7in iyile\u015ftirici sonu\u00e7lar sunma potansiyeline sahiptir.<\/p>\n

Dr. McCray, \"Emily's Entourage'dan kistik fibrozis i\u00e7in daha etkili ve hedefe y\u00f6nelik tedaviler geli\u015ftirmeye y\u00f6nelik ara\u015ft\u0131rma \u00e7abalar\u0131m\u0131z\u0131 destekleyecek bu c\u00f6mert hibeyi almaktan onur duyuyoruz\" dedi. \"Ara\u015ft\u0131rmam\u0131z, terap\u00f6tiklerin hava yolu epitel h\u00fccrelerine nas\u0131l iletildi\u011fini geli\u015ftirerek KF tedavisinde kar\u015f\u0131lanmam\u0131\u015f kritik ihtiyac\u0131 ele almay\u0131 ama\u00e7l\u0131yor - bu, KF'li ki\u015filerin son 10%'si i\u00e7in tedavileri ilerletmede \u00f6nemli bir ad\u0131m.\"<\/p>\n

Projenin iki y\u00f6nl\u00fc hedefleri aras\u0131nda, insan hava yolu epiteline kargo teslimi i\u00e7in en verimli varyantlar\u0131 belirlemek \u00fczere kapsid taramas\u0131n\u0131 tamamlamak ve terap\u00f6tik kargo teslimi i\u00e7in \u00f6nde gelen kapsidlerin etkinli\u011fini de\u011ferlendirmek yer al\u0131yor. Ara\u015ft\u0131rma ekibi, en son teknolojilerden ve disiplinler aras\u0131 i\u015fbirli\u011finden yararlanarak, KF ile ya\u015fayan bireyler i\u00e7in t\u00fcr\u00fcn\u00fcn ilk \u00f6rne\u011fi olan hedefe y\u00f6nelik bir tedavi se\u00e7ene\u011fi geli\u015ftirmek amac\u0131yla bu heyecan verici bilimi ilerletmeye haz\u0131rlan\u0131yor.<\/p>\n

Emily's Entourage'\u0131n ba\u015f bilimsel sorumlusu Chandra Ghose, \"Emily's Entourage, toplumun 100%'sine ula\u015fma hedefiyle KF'li ki\u015filerin tedavisine y\u00f6nelik yeni yakla\u015f\u0131mlar\u0131 ke\u015ffeden yenilik\u00e7i ara\u015ft\u0131rma giri\u015fimlerini desteklemekten gurur duyuyor\" dedi. \"Yeni \u0130\u015fbirli\u011fi Hibe program\u0131m\u0131z arac\u0131l\u0131\u011f\u0131yla verilen bu a\u00e7\u0131l\u0131\u015f \u00f6d\u00fcl\u00fc, KF tedavisindeki at\u0131l\u0131mlar\u0131 h\u0131zland\u0131rma ve bu hastal\u0131ktan etkilenen t\u00fcm bireyler i\u00e7in hayat de\u011fi\u015ftiren tedavilerin geli\u015ftirilmesini h\u0131zland\u0131rma taahh\u00fcd\u00fcm\u00fcz\u00fcn alt\u0131n\u0131 \u00e7izen \u00f6nemli bir kilometre ta\u015f\u0131na i\u015faret ediyor.\"<\/p>\n

EE, mevcut CFTR mod\u00fclat\u00f6rlerinden yararlanamayan KF pop\u00fclasyonunun son 10%'si i\u00e7in h\u0131zland\u0131r\u0131lm\u0131\u015f, d\u00f6n\u00fc\u015ft\u00fcr\u00fcc\u00fc tedavilere yol a\u00e7an ve hi\u00e7 kimsenin geride kalmamas\u0131n\u0131 sa\u011flayan en son ara\u015ft\u0131rmalar\u0131 desteklemeye kararl\u0131d\u0131r.<\/p>\n

Emily's Entourage'\u0131n (EE) Hibe Program\u0131 Hakk\u0131nda\u00a0<\/b><\/p>\n

Emily's Entourage'\u0131n (EE) Hibe Program\u0131, mevcut mutasyon hedefli tedavilerden faydalanamayan kistik fibrozis (KF) hastalar\u0131 i\u00e7in ara\u015ft\u0131rma ve terap\u00f6tik geli\u015ftirmeyi h\u0131zland\u0131rmak amac\u0131yla hibe fonu sa\u011flamaktad\u0131r. EE, \u00e7eviri hibeleri, i\u015fbirli\u011fine dayal\u0131 hibeler ve klinik \u00f6ncesi ke\u015fif hibeleri dahil olmak \u00fczere \u00e7e\u015fitli hibe finansman mekanizmalar\u0131n\u0131n yan\u0131 s\u0131ra giri\u015fimci hay\u0131rseverlik yat\u0131r\u0131mlar\u0131 yoluyla da finansman sa\u011flamaktad\u0131r.<\/p>\n

EE bug\u00fcne kadar d\u00fcnyan\u0131n d\u00f6rt bir yan\u0131ndaki \u00e7ok disiplinli ekiplere milyonlarca dolar hibe vermi\u015f ve milyonlarca dolarl\u0131k devam finansman\u0131n\u0131n sa\u011flanmas\u0131na yard\u0131mc\u0131 olmu\u015ftur. Verilen hibeleri g\u00f6r\u00fcnt\u00fclemek i\u00e7in \u015fu adresi ziyaret edin https:\/\/www.emilysentourage.org\/awarded-grants\/<\/a>. EE'nin finansman f\u0131rsatlar\u0131 hakk\u0131nda daha fazla bilgi edinmek i\u00e7in \u015fu adresi ziyaret edin https:\/\/www.emilysentourage.org\/funding-opportunities\/<\/a>.<\/p>\n

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

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 sat\u0131n al\u0131nan bir KF gen terapisi \u015firketini kurmu\u015f, klinik deneylere kat\u0131l\u0131m\u0131 h\u0131zland\u0131rmak i\u00e7in bir hasta kay\u0131t defteri ve klinik deney e\u015fle\u015ftirme program\u0131 geli\u015ftirmi\u015f ve y\u00fcksek etkili ara\u015ft\u0131rma ve ila\u00e7 geli\u015ftirmeye y\u00f6nelik d\u00fcnya \u00e7ap\u0131nda \u00e7abalara \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 emilysentourage.org<\/a>.<\/p>\n

 <\/p>\n

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

Hannah Kleckner<\/p>\n

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

hannah@emilysentourage.org<\/a><\/p>","protected":false},"excerpt":{"rendered":"

Iowa \u00dcniversitesi, CHOP ve Rochester \u00dcniversitesi'nden ara\u015ft\u0131rma ekipleri, EE'den yeni sa\u011flanan fon sayesinde, geli\u015fmi\u015f yeni adeno-ili\u015fkili vir\u00fcs (AAV) kapsid varyantlar\u0131ndan yararlanarak mevcut kistik fibroz tedavilerindeki mevcut s\u0131n\u0131rlamalar\u0131n \u00fcstesinden gelmeyi ama\u00e7l\u0131yor. Lower Merion, PA - 29 May\u0131s 2024 - Emily's Entourage (EE), kistik fibrozis tedavisini h\u0131zland\u0131ran yenilik\u00e7i bir 501(c)3... <\/p>\n

<\/div>\n

Daha fazla bilgi edinin<\/a><\/p>","protected":false},"author":1719,"featured_media":21085,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_seopress_titles_title":"%%post_title%%","_seopress_titles_desc":"%%post_excerpt%%","_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":"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/2024-Collaboration-Grant-Award-Facebook.png","_seopress_social_fb_img_attachment_id":21086,"_seopress_social_fb_img_width":940,"_seopress_social_fb_img_height":788,"_seopress_social_twitter_title":"","_seopress_social_twitter_desc":"","_seopress_social_twitter_img":"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/2024-Collaborative-Grant-Award-XLI.png","_seopress_social_twitter_img_attachment_id":21087,"_seopress_social_twitter_img_width":1600,"_seopress_social_twitter_img_height":900,"_seopress_redirections_value":"","_seopress_redirections_enabled":"","_seopress_redirections_enabled_regex":"","_seopress_redirections_logged_status":"both","_seopress_redirections_param":"","_seopress_redirections_type":301,"_seopress_analysis_target_kw":"","_seopress_news_disabled":"","_seopress_video_disabled":"","_seopress_video":[],"_seopress_pro_schemas_manual":[],"_seopress_pro_rich_snippets_disable_all":"","_seopress_pro_rich_snippets_disable":[],"_seopress_pro_schemas":[],"_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":[7,188,78,111],"class_list":["post-21076","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-press-releases","tag-cystic-fibrosis","tag-gene-therapy","tag-nonsense-mutations","tag-research"],"acf":[],"jetpack_sharing_enabled":true,"jetpack_featured_media_url":"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/McCray-Release-Featured-Image-May-2024.png","post_mailing_queue_ids":[],"_links":{"self":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/21076","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=21076"}],"version-history":[{"count":5,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/21076\/revisions"}],"predecessor-version":[{"id":21091,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/21076\/revisions\/21091"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media\/21085"}],"wp:attachment":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media?parent=21076"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/categories?post=21076"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/tags?post=21076"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}