{"id":16623,"date":"2022-02-28T09:00:11","date_gmt":"2022-02-28T14:00:11","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?p=16623"},"modified":"2022-02-28T08:58:26","modified_gmt":"2022-02-28T13:58:26","slug":"440000-awarded-in-round-6-funding-for-cystic-fibrosis-research","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/tr\/440000-awarded-in-round-6-funding-for-cystic-fibrosis-research\/","title":{"rendered":"EE, Kistik Fibrozis Ara\u015ft\u0131rmalar\u0131 i\u00e7in 6. Tur Finansman\u0131nda $440,000 \u00d6d\u00fcl Verdi"},"content":{"rendered":"

Emily's Entourage'\u0131n (EE) kistik fibrozis (KF) ara\u015ft\u0131rmalar\u0131n\u0131 ve ila\u00e7 geli\u015ftirmeyi ilerletmek amac\u0131yla iki yeni hibe i\u00e7in $440.000 fon sa\u011flad\u0131\u011f\u0131n\u0131 payla\u015fmaktan heyecan duyuyoruz. Hibeler, yeni bir anlams\u0131z mutasyon gelincik modelinin ve lipid nanopartik\u00fcller kullan\u0131larak yeni bir gen terapisi formunun geli\u015ftirilmesini desteklemektedir.\u00a0<\/span><\/p>\n

\n

\"Bu hibeler, Emily's Entourage'\u0131n kritik kar\u015f\u0131lanmam\u0131\u015f ihtiya\u00e7lar\u0131 kar\u015f\u0131lamak i\u00e7in hayat kurtaran ara\u015ft\u0131rmalar\u0131 ilerletme ve \u00f6zellikle mevcut mutasyon hedefli tedavilerden yararlanamayan KF toplulu\u011fu i\u00e7in umut verici tedavi f\u0131rsatlar\u0131n\u0131 h\u0131zl\u0131 bir \u015fekilde takip etme konusundaki sars\u0131lmaz kararl\u0131l\u0131\u011f\u0131n\u0131 yans\u0131t\u0131yor.<\/span>\u201c<\/span><\/strong><\/p>\n

- Chandra Ghose, PhD, Ba\u015f Bilim Sorumlusu, Emily's Entourage<\/strong><\/span><\/p>\n<\/blockquote>\n

HIBE ALICILARI:<\/strong><\/h2>\n
\n

Anlams\u0131z mutasyona y\u00f6nelik tedavileri incelemek i\u00e7in W1286X kistik fibrozis gelincik modeli olu\u015fturma
\n<\/b><\/h3>\n

\"HeadshotXingshen Sun, PhD
\n<\/span>Iowa \u00dcniversitesi<\/span><\/i><\/p>\n

Bir ila\u00e7 veya tedavi yakla\u015f\u0131m\u0131 insan klinik deneylerinde kullan\u0131lmadan \u00f6nce, g\u00fcvenlik ve etkinli\u011fini de\u011ferlendirmek i\u00e7in \u00f6ncelikle bir hayvan modelinde test edilmesi gerekir. Ancak, bug\u00fcne kadar kistik fibroz transmembran iletkenlik reg\u00fclat\u00f6r\u00fc (CFTR) sa\u00e7ma mutasyonuna sahip uygun bir hayvan modeli mevcut de\u011fildir. \u00d6nceki \u00e7al\u0131\u015fmalar, gelinci\u011fin solunum yetmezli\u011fine yol a\u00e7an bir akci\u011fer hastal\u0131\u011f\u0131 geli\u015ftirmesi ve KF ile ili\u015fkili diyabete yol a\u00e7an pankreas hastal\u0131klar\u0131 geli\u015ftirmesi nedeniyle bir KF modeli i\u00e7in ideal bir t\u00fcr oldu\u011funu g\u00f6stermektedir. Bu proje, insan W1282X mutasyonuna benzer \u015fekilde CFTR geninde W1286X mutasyonunu i\u00e7eren bir KF gelincik modeli olu\u015fturmay\u0131 ama\u00e7layacakt\u0131r. Gelincik modeli, birden fazla ila\u00e7 kullan\u0131larak yap\u0131lan tedavinin gelincikteki hastal\u0131k semptomlar\u0131n\u0131 azalt\u0131p azaltamayaca\u011f\u0131n\u0131 test etmek i\u00e7in kullan\u0131lacakt\u0131r. Bu yeni W1286X gelincik modeli, etkili ve g\u00fcvenli terap\u00f6tik yakla\u015f\u0131mlar\u0131n belirlenmesini h\u0131zland\u0131rmak i\u00e7in yeni bir f\u0131rsat sunarak, CFTR-W1282X mutasyonu ve potansiyel olarak di\u011fer nonsense mutasyonlar\u0131 olan hastalar i\u00e7in yeni ila\u00e7lar\u0131n veya yeni terap\u00f6tik stratejilerin belirlenme s\u00fcrecini, h\u00fccre tabanl\u0131 sistemlerin veya kemirgen modellerinin izin vermeyece\u011fi \u015fekilde \u00f6nemli \u00f6l\u00e7\u00fcde k\u0131saltmaktad\u0131r.<\/span><\/p>\n

\n

W1282X'i hedefleyen baz d\u00fczenleyicilerin pulmoner viral olmayan iletimi
\n<\/b><\/h3>\n

\"HeadshotDebadyuti (Rana) Ghosh, PhD (resimde) ve Hugh Smyth, PhD
\n<\/span>Austin'deki Teksas \u00dcniversitesi<\/span><\/i><\/p>\n

Gen d\u00fczenleme terapisi<\/span>o m<\/span>KF ile ili\u015fkili kusurlu geni onarabilmek, hastal\u0131k i\u00e7in kal\u0131c\u0131 bir tedavi elde etme potansiyeline sahiptir. \u015eu anda mutasyonu etkili bir \u015fekilde d\u00fczeltebilen ve normal h\u00fccre ve akci\u011fer fonksiyonuyla ilgili anahtar proteini geri y\u00fckleyebilen bir teknoloji bulunmaktad\u0131r. Ancak bu tedaviyi v\u00fccutta bozulmadan hastaya ula\u015ft\u0131rmak zordur. Bu proje, gen terapisi kargosunu bozunmaya kar\u015f\u0131 koruyabilecek ve nihayetinde akci\u011fere ula\u015ft\u0131r\u0131labilecek farmas\u00f6tik ta\u015f\u0131y\u0131c\u0131lar\u0131 - veya gen terapisi kargosunu iletmek i\u00e7in kullan\u0131lan malzemeleri - tarayacak ve tan\u0131mlayacakt\u0131r.<\/span>gs <\/span>inhale edilen ila\u00e7lar olarak.<\/span><\/p>\n

 <\/p>\n

 <\/p>\n

\n

EE, 2011 y\u0131l\u0131ndan bu yana d\u00fcnya genelinde 24 ara\u015ft\u0131rma projesine $5.2 milyondan fazla hibe vermi\u015ftir. Bu son hibeler, KF'nin nonsense mutasyonlar\u0131na sahip olanlar da dahil olmak \u00fczere mevcut mutasyon hedefli tedavilerden fayda g\u00f6rmeyen KF'li ki\u015filer i\u00e7in terap\u00f6tik geli\u015fimi h\u0131zland\u0131rmak amac\u0131yla EE taraf\u0131ndan verilen hibe fonunun alt\u0131nc\u0131 turunu olu\u015fturmaktad\u0131r.\u00a0<\/span><\/p>\n

Hibe alanlara ve KF'li t\u00fcm insanlar i\u00e7in kar\u015f\u0131lanmam\u0131\u015f kritik ihtiya\u00e7lar\u0131 h\u0131zl\u0131 bir \u015fekilde kar\u015f\u0131lamaya \u00f6nc\u00fcl\u00fck eden t\u00fcm adanm\u0131\u015f bilim insanlar\u0131na minnettar\u0131z - kimse geride kalmas\u0131n! Verilen t\u00fcm ara\u015ft\u0131rma hibelerinin kapsaml\u0131 bir listesini g\u00f6r\u00fcnt\u00fcleyin <\/span>Burada<\/span><\/a>.\u00a0<\/span><\/p>","protected":false},"excerpt":{"rendered":"

Emily's Entourage'\u0131n (EE) kistik fibrozis (KF) ara\u015ft\u0131rmalar\u0131n\u0131 ve ila\u00e7 geli\u015ftirmeyi ilerletmek amac\u0131yla iki yeni hibe i\u00e7in $440.000 fon sa\u011flad\u0131\u011f\u0131n\u0131 payla\u015fmaktan heyecan duyuyoruz. Hibeler, yeni bir anlams\u0131z mutasyon gelincik modelinin ve lipid nanopartik\u00fcller kullan\u0131larak yeni bir gen terapisi formunun geli\u015ftirilmesini desteklemektedir. \u201cBu hibeler Emily's Entourage'\u0131n teredd\u00fcts\u00fcz deste\u011fini yans\u0131t\u0131yor... <\/p>\n

<\/div>\n

Daha fazla bilgi edinin<\/a><\/p>","protected":false},"author":1281,"featured_media":16748,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_seopress_titles_title":"EE Awards $440,000 in Round 6 Funding for Cystic Fibrosis Research","_seopress_titles_desc":"We are thrilled to share that Emily\u2019s Entourage (EE) has awarded $440,000 in funding for two new grants to advance cystic fibrosis (CF) research and drug development.","_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":"","_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":"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_type":"none"}],"_seopress_pro_rich_snippets_disable_all":"","_seopress_pro_rich_snippets_disable":[],"_seopress_pro_schemas":[],"_monsterinsights_skip_tracking":false,"_monsterinsights_sitenote_active":false,"_monsterinsights_sitenote_note":"","_monsterinsights_sitenote_category":0,"_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":[1],"tags":[],"class_list":["post-16623","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-uncategorized"],"acf":[],"jetpack_featured_media_url":"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/Grantee-Blog-Feature-1-1.png","jetpack_sharing_enabled":true,"post_mailing_queue_ids":[],"_links":{"self":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/16623","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\/1281"}],"replies":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/comments?post=16623"}],"version-history":[{"count":5,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/16623\/revisions"}],"predecessor-version":[{"id":16629,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/16623\/revisions\/16629"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media\/16748"}],"wp:attachment":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media?parent=16623"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/categories?post=16623"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/tags?post=16623"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}