{"id":27114,"date":"2026-09-09T14:51:00","date_gmt":"2026-09-09T18:51:00","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?p=27114"},"modified":"2026-09-09T14:52:12","modified_gmt":"2026-09-09T18:52:12","slug":"emilys-entourage-breaks-new-ground-with-ai-powered-grant-to-identify-next-generation-cf-gene-therapy-delivery-systems","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/tr\/emilys-entourage-breaks-new-ground-with-ai-powered-grant-to-identify-next-generation-cf-gene-therapy-delivery-systems\/","title":{"rendered":"Emily\u2019s Entourage, Yeni Nesil Kistik Fibrozis Gen Tedavisi Da\u011f\u0131t\u0131m Sistemlerini Belirlemek \u0130\u00e7in Yapay Zeka Destekli Hibeyle \u00c7\u0131\u011f\u0131r A\u00e7\u0131yor"},"content":{"rendered":"

Yapay zeka destekli lipit nanopartik\u00fcl taramas\u0131, solunum yoluyla verilen Kistik Fibrozis gen tedavisindeki uzun s\u00fcredir devam eden engelleri a\u015fmay\u0131 ama\u00e7l\u0131yor<\/span><\/i><\/p>\n

\"\"

Emily's Entourage (EE), Massachusetts Teknoloji Enstit\u00fcs\u00fc'ndeki (MIT) Anderson Laboratuvar\u0131'nda Doktora Sonras\u0131 Ara\u015ft\u0131rmac\u0131 olan Dr. Jacob Witten'a verilen 2026 y\u0131l\u0131na ait alt\u0131nc\u0131 ve son \u00c7eviri Ara\u015ft\u0131rmas\u0131 Hibesini duyurdu.<\/em><\/p><\/div>\n

Lower Merion, PA\u20149 Eyl\u00fcl 2026\u2014Mevcut 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\u2019s Entourage (EE), Massachusetts Teknoloji Enstit\u00fcs\u00fc'ndeki (MIT) Anderson Laboratuvar\u0131'nda Doktora Sonras\u0131 Ara\u015ft\u0131rma G\u00f6revlisi olan Dr. Jacob Witten'a verilen 2026 y\u0131l\u0131ndaki alt\u0131nc\u0131 ve son \u00c7eviri Ara\u015ft\u0131rma Hibesini duyurmaktan gurur duyar.<\/span><\/p>\n

Dr. Witten'in projesi, mevcut mutasyon odakl\u0131 tedavilerden yararlanmayan KF hastalar\u0131nda CFTR fonksiyonunu yeniden kazand\u0131rman\u0131n \u00f6n\u00fcndeki b\u00fcy\u00fck bir engeli a\u015farak, \u00f6zellikle solunum yoluyla al\u0131nan gen tedavisi i\u00e7in tasarlanm\u0131\u015f geli\u015fmi\u015f lipit nanopartik\u00fcller (LNP'ler) geli\u015ftirmeyi ama\u00e7lamaktad\u0131r.<\/span><\/p>\n

\u201cSon 10%\u201d olarak bilinen bu ki\u015filer i\u00e7in, gen terapisi veya gen d\u00fczenleme yoluyla CFTR aktivitesini geri kazand\u0131rmak, en umut verici potansiyel tedavi stratejilerinden birini olu\u015fturmaktad\u0131r. Ancak, on y\u0131llard\u0131r s\u00fcren klinik denemeler, genetik tedavilerin akci\u011ferlere g\u00fcvenli ve etkili bir \u015fekilde ula\u015ft\u0131r\u0131lmas\u0131n\u0131n h\u00e2l\u00e2 b\u00fcy\u00fck bir zorluk oldu\u011funu ortaya koymu\u015ftur.<\/span><\/p>\n

Lipid nanopartik\u00fclleri, COVID-19 a\u015f\u0131lar\u0131 ve genetik hastal\u0131klar\u0131n tedavileri dahil olmak \u00fczere mRNA tabanl\u0131 tedaviler i\u00e7in yak\u0131n zamanda g\u00fc\u00e7l\u00fc ta\u015f\u0131y\u0131c\u0131 ara\u00e7lar olarak ortaya \u00e7\u0131km\u0131\u015ft\u0131r. Ancak bu ba\u015far\u0131n\u0131n akci\u011fere uyarlanmas\u0131, kal\u0131n mukus, s\u0131n\u0131rl\u0131 h\u00fccresel al\u0131m ve havayolu h\u00fccreleri aras\u0131ndaki s\u0131k\u0131 ba\u011flant\u0131lar dahil olmak \u00fczere havayolunun kendine \u00f6zg\u00fc biyolojik bariyerleri nedeniyle zor oldu\u011funu kan\u0131tlam\u0131\u015ft\u0131r.<\/span><\/p>\n

Dr. Witten'in ara\u015ft\u0131rmas\u0131, \u00f6zellikle havayolu yoluyla iletim i\u00e7in optimize edilmi\u015f yeni LNP form\u00fclasyonlar\u0131 tasarlay\u0131p tarayarak bu zorluklar\u0131n \u00fcstesinden gelmeyi ama\u00e7lamaktad\u0131r. Ekibi, g\u00fcvenli\u011fi art\u0131rmak i\u00e7in biyobozunur lipit bile\u015fenleri geli\u015ftirecek, umut vadeden form\u00fclasyonlar\u0131 belirlemek i\u00e7in yapay zekadan (AI) yararlanacak ve verimli h\u00fccre al\u0131m\u0131 ile mukus penetrasyonunu art\u0131rmak i\u00e7in y\u00fczey kimyas\u0131n\u0131 optimize edecektir.<\/span><\/p>\n

Projenin \u00f6nemli bir inovasyonu, insan hava yolu biyolojisini yak\u0131ndan taklit eden geli\u015fmi\u015f laboratuvar modelleri olan, apical-out hava yolu organoidlerinin kullan\u0131m\u0131d\u0131r. Bu modeller, \u00e7ok say\u0131da LNP form\u00fclasyonunun h\u0131zl\u0131 ve y\u00fcksek verimli taranmas\u0131n\u0131 sa\u011flayarak akci\u011fere \u00f6zel olarak uyarlanm\u0131\u015f ta\u015f\u0131y\u0131c\u0131 sistemlerin ke\u015ffini h\u0131zland\u0131rmaktad\u0131r.<\/span><\/p>\n

Ekip, yapay zeka destekli lipid tasar\u0131m\u0131n\u0131 fizyolojik olarak ilgili solunum yolu modelleri ile birle\u015ftirerek, soluma yoluyla iletim i\u00e7in hem daha etkili hem de daha iyi tolere edilen LNP'leri belirlemeyi ama\u00e7lamaktad\u0131r. En umut vadeden adaylar daha sonra Kistik Fibrozis (CF) solunum yolu k\u00fclt\u00fcrleri ve insan akci\u011fer eksplantlar\u0131 dahil olmak \u00fczere ek translasyonel modellerde de\u011ferlendirilecektir.<\/span><\/p>\n

\u201cKF gen tedavisindeki en b\u00fcy\u00fck zorluklardan biri, tedavileri akci\u011ferdeki do\u011fru h\u00fccrelere ula\u015ft\u0131rmakt\u0131r\u201d dedi Dr. Witten. \u201cBu \u00e7al\u0131\u015fma, \u015fu anda tedavi se\u00e7eneklerinden yoksun olan ki\u015filer i\u00e7in daha etkili inhale genetik tedavilerin \u00f6n\u00fcn\u00fc a\u00e7mak amac\u0131yla \u00f6zellikle solunum yolu i\u00e7in tasarlanm\u0131\u015f ta\u015f\u0131y\u0131c\u0131 sistemler geli\u015ftirmeye odaklan\u0131yor.\u201d<\/span><\/p><\/blockquote>\n

Son a\u015famadaki 10% denemesine kat\u0131lan ki\u015filer i\u00e7in, mRNA veya gen d\u00fczenleme ara\u00e7lar\u0131n\u0131n etkili bir \u015fekilde uygulanmas\u0131, akci\u011fer fonksiyonunun geri kazan\u0131lmas\u0131na y\u00f6nelik d\u00f6n\u00fc\u015ft\u00fcr\u00fcc\u00fc bir ad\u0131m olabilir.<\/span><\/p>\n

\u201cKistik fibrozis i\u00e7in n\u00fckleotit bazl\u0131 tedavilerin t\u00fcm potansiyelini ortaya \u00e7\u0131karmak ad\u0131na havayolu ile iletim zorlu\u011funun \u00e7\u00f6z\u00fclmesi hayati \u00f6nem ta\u015f\u0131yor,\u201d dedi EE'nin Bilim Direkt\u00f6r\u00fc PhD Chandra Ghose. \u201cYapay zekay\u0131 geli\u015fmi\u015f havayolu modelleriyle birle\u015ftiren yenilik\u00e7i stratejileri destekleyerek, mevcut CFTR mod\u00fclat\u00f6r tedavilerinden yararlanamayanlar i\u00e7in \u00f6zel olarak tasarlanm\u0131\u015f iletim teknolojilerinin geli\u015ftirilmesini h\u0131zland\u0131rmaya yard\u0131mc\u0131 oluyoruz.\u201d<\/span><\/p><\/blockquote>\n

 <\/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 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

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

Emily\u2019s Entourage (EE), mevcut mutasyona y\u00f6nelik 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, klinik denemelere kat\u0131l\u0131m\u0131 h\u0131zland\u0131rmak i\u00e7in k\u00fcresel bir hasta veritaban\u0131 ve klinik deneme e\u015fle\u015ftirme program\u0131 geli\u015ftirmi\u015f ve y\u00fcksek etkili ara\u015ft\u0131rma ve ila\u00e7 geli\u015ftirme \u00e7al\u0131\u015fmalar\u0131n\u0131 te\u015fvik etmek amac\u0131yla d\u00fcnya \u00e7ap\u0131nda giri\u015fimlere \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<\/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\u00a0

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

AI-guided lipid nanoparticle screening aims to overcome longstanding barriers to inhaled CF gene therapy Lower Merion, PA\u2014September 9, 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 sixth and final 2026 Translational Research Grant awarded to… <\/p>\n

<\/div>\n

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