{"id":20025,"date":"2023-11-21T09:48:14","date_gmt":"2023-11-21T14:48:14","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?p=20025"},"modified":"2023-12-20T05:15:32","modified_gmt":"2023-12-20T10:15:32","slug":"emilys-entourage-awards-220000-research-grant-to-dr-alexandra-pitrowski-daspit-of-the-university-of-michigan-to-support-non-viral-nucleic-acid-delivery-research-for-cf-nonsense-mutations","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/tr\/emilys-entourage-awards-220000-research-grant-to-dr-alexandra-pitrowski-daspit-of-the-university-of-michigan-to-support-non-viral-nucleic-acid-delivery-research-for-cf-nonsense-mutations\/","title":{"rendered":"Emily's Entourage, Michigan \u00dcniversitesi'nden Dr. Alexandra Pitrowski-Daspit'e KF Nonsense Mutasyonlar\u0131 i\u00e7in Viral Olmayan N\u00fckleik Asit \u0130letimi Ara\u015ft\u0131rmalar\u0131n\u0131 Desteklemek \u00dczere $220,000 Ara\u015ft\u0131rma Bursu Verdi"},"content":{"rendered":"

Ara\u015ft\u0131rma \u015fu konulara odaklanm\u0131\u015ft\u0131r <\/span><\/i>KF tedavisi i\u00e7in n\u00fckleik asit bazl\u0131 terap\u00f6tiklerin v\u00fccuttaki hedef dokulara ve h\u00fccrelere iletimini iyile\u015ftirmek i\u00e7in polimerik ara\u00e7lar geli\u015ftirmek.\u00a0\u00a0<\/span><\/i><\/p>\n

Lower Merion, PA - 20 Kas\u0131m<\/b>, 2023<\/b> - Kistik fibrozis (KF) hastalar\u0131n\u0131n son 10%'si i\u00e7in yeni tedaviler ve tedavi ara\u015ft\u0131rmalar\u0131n\u0131 h\u0131zland\u0131ran yenilik\u00e7i bir 501(c)3 kurulu\u015fu olan Emily's Entourage (EE), bug\u00fcn <\/span>$220,000<\/span> Dr. Alexandra S. Piotrowski-Daspit, PhD, Michigan \u00dcniversitesi'nde Biyomedikal M\u00fchendisli\u011fi ve \u0130\u00e7 Hastal\u0131klar\u0131, Pulmoner ve Yo\u011fun Bak\u0131m T\u0131bb\u0131 B\u00f6l\u00fcm\u00fc'nde Yard\u0131mc\u0131 Do\u00e7ent. Hibe<\/span> KF'nin nonsense mutasyonlar\u0131na sahip ki\u015filer i\u00e7in daha etkili tedavi se\u00e7enekleri sa\u011flamak \u00fczere viral olmayan n\u00fckleik asit iletimine odaklanan ara\u015ft\u0131rmalar\u0131 desteklemektedir.<\/span><\/p>\n

\"\"

Alexandra Piotrowski-Daspit, PhD
Michigan \u00dcniversitesi<\/p><\/div>\n

KF, kistik fibroz transmembran iletkenlik reg\u00fclat\u00f6r\u00fc (CFTR) genindeki mutasyonlar\u0131n neden oldu\u011fu ilerleyici bir genetik bozukluktur. CFTR'de ortaya \u00e7\u0131kan i\u015flevsel de\u011fi\u015fikliklere g\u00f6re s\u0131n\u0131fland\u0131r\u0131lan 1.700'den fazla KF'ye neden olan mutasyon vard\u0131r. W1282X ve G542X gibi sa\u00e7ma mutasyonlar erken stop kodonlar\u0131na neden olarak kesilmi\u015f CFTR proteini ve i\u015flev kayb\u0131 \u00fcretir. Gen d\u00fczenleme, gen terapisi ve mRNA terapisi dahil olmak \u00fczere n\u00fckleik asit bazl\u0131 tedaviler, i\u015flevsiz CFTR'yi de\u011fi\u015ftirme veya d\u00fczeltme potansiyeli sunmaktad\u0131r. Bununla birlikte, bu tedavilerin hedef organlara ula\u015ft\u0131r\u0131lmas\u0131, klinik \u00e7evirinin \u00f6n\u00fcndeki birincil engel olmaya devam etmektedir.\u00a0<\/span><\/p>\n

Proje, \"<\/span>KF nonsense mutasyonlar\u0131 i\u00e7in \u00e7oklu organ hedefli viral olmayan n\u00fckleik asit iletimi<\/span><\/i>\"n\u00fckleik asit bazl\u0131 terap\u00f6tiklerin ba\u015fta akci\u011fer ve gastrointestinal sistem olmak \u00fczere v\u00fccuttaki hedef doku ve h\u00fccrelere iletimini iyile\u015ftirmek i\u00e7in polimerik ara\u00e7lar\u0131n geli\u015ftirilmesini ara\u015ft\u0131rmay\u0131 ama\u00e7lamaktad\u0131r.\u00a0<\/span><\/p>\n

\"N\u00fckleik asit bazl\u0131 terap\u00f6tikler, i\u015flevsiz CFTR'yi de\u011fi\u015ftirmek veya d\u00fczeltmek i\u00e7in \u00e7al\u0131\u015fma \u015fekilleri nedeniyle \u00f6zellikle umut vericidir. Bu tedavi yakla\u015f\u0131mlar\u0131 KF'li insanlar i\u00e7in tedavi se\u00e7eneklerine yol a\u00e7abilir, ancak bunlar\u0131n etkili bir \u015fekilde sunulabilece\u011finden emin olmam\u0131z gerekiyor,\" diyor EE Bilimsel Ba\u015f Sorumlusu PhD Chandra Ghose. \"Nadir ve anlams\u0131z mutasyonlara sahip olanlar da dahil olmak \u00fczere t\u00fcm KF'li insanlar i\u00e7in tedaviler geli\u015ftirmek amac\u0131yla ara\u00e7 kutumuza eklemeler yapmaya ve yenilikler yapmaya devam ederken bu alandaki ara\u015ft\u0131rma ve geli\u015ftirme \u00e7abalar\u0131 kritik \u00f6nem ta\u015f\u0131yacakt\u0131r.\"\u00a0<\/span><\/p>\n

Hibe, ara\u015ft\u0131rma ekibinin i\u015flevsiz CFTR'yi de\u011fi\u015ftirmek veya d\u00fczeltmek i\u00e7in genom d\u00fczenleme ajanlar\u0131na odaklanarak n\u00fckleik asit bazl\u0131 terap\u00f6tikleri ke\u015ffetme \u00e7abalar\u0131n\u0131 destekleyecektir. Nihai ama\u00e7, hedeflenen tedaviler i\u00e7in etkili ara\u00e7lar geli\u015ftirmektir. <\/span>in vivo <\/span><\/i>W1282X KF sa\u00e7ma mutasyonunun yaratt\u0131\u011f\u0131 benzersiz zorluklar\u0131 ele alarak, akci\u011ferler ve gastrointestinal sistemdeki epitellere terap\u00f6tik n\u00fckleik asit iletimi.<\/span><\/p>\n

Dr. Piotrowski-Daspit, \"Emily's Entourage'a, n\u00fckleik asit bazl\u0131 terap\u00f6tiklerin hedefe y\u00f6nelik da\u011f\u0131t\u0131m\u0131na y\u00f6nelik polimerik ara\u00e7lar konusundaki ara\u015ft\u0131rmam\u0131z\u0131 ilerletmemizi sa\u011flayacak bu c\u00f6mert hibe i\u00e7in inan\u0131lmaz derecede minnettar\u0131z\" dedi. \"\u00c7al\u0131\u015fmalar\u0131m\u0131z\u0131n uzun vadeli hedefi, KF'li t\u00fcm insanlar i\u00e7in uygulanabilir bir tedavi geli\u015ftirerek onlara eksiksiz ve \u00fcretken bir ya\u015fam s\u00fcrme f\u0131rsat\u0131 sunmakt\u0131r.\"<\/span><\/p>\n

\u00d6nerilen disiplinler aras\u0131 ara\u015ft\u0131rma, mevcut CFTR mod\u00fclat\u00f6r tedavilerinden yararlanamayan nadir ve anlams\u0131z mutasyonlara sahip KF hasta pop\u00fclasyonunun son 10%'si de dahil olmak \u00fczere, KF'li insanlar i\u00e7in umut verici n\u00fckleik asit terap\u00f6tiklerinin \u00e7evirisini ilerleterek klinik a\u00e7\u0131dan olduk\u00e7a \u00f6nemlidir.\u00a0<\/span><\/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.<\/span><\/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 <\/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>.\u00a0<\/span><\/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 <\/span>emilysentourage.org<\/span><\/a>.<\/span><\/p>\n

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

Emily'nin \u00c7evresi<\/span><\/p>\n

admin@emilysentourage.org<\/span><\/a>\u00a0<\/span><\/p>","protected":false},"excerpt":{"rendered":"

Ara\u015ft\u0131rma, n\u00fckleik asit bazl\u0131 terap\u00f6tiklerin KF tedavisi i\u00e7in v\u00fccuttaki hedef doku ve h\u00fccrelere iletimini iyile\u015ftirmek i\u00e7in polimerik ara\u00e7lar geli\u015ftirmeye odaklanm\u0131\u015ft\u0131r. Lower Merion, PA - 20 Kas\u0131m 2023 - Emily's Entourage (EE), yeni tedaviler i\u00e7in ara\u015ft\u0131rmalar\u0131 h\u0131zland\u0131ran ve KF hastal\u0131\u011f\u0131na \u00e7are bulan yenilik\u00e7i bir 501(c)3... <\/p>\n

<\/div>\n

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