{"id":17522,"date":"2022-11-03T11:41:28","date_gmt":"2022-11-03T15:41:28","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?page_id=17522"},"modified":"2023-12-20T05:15:53","modified_gmt":"2023-12-20T10:15:53","slug":"emilys-entourage-awards-research-grant-to-team-exploring-novel-gene-editing-technology","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/tr\/emilys-entourage-awards-research-grant-to-team-exploring-novel-gene-editing-technology\/","title":{"rendered":"Emily's Entourage Yeni Gen D\u00fczenleme Teknolojisini Ke\u015ffeden Ekibe Ara\u015ft\u0131rma Bursu Verdi"},"content":{"rendered":"

Proje, belirli CFTR mutasyonlar\u0131n\u0131 g\u00fcvenli ve verimli bir \u015fekilde d\u00fczeltmek i\u00e7in prime editing ad\u0131 verilen gen d\u00fczenleme teknolojisinden yararlanmaya odaklanacak ve bu d\u00fczenleme kargosunu vir\u00fcs benzeri partik\u00fcller (VLP) yoluyla iletme olas\u0131l\u0131\u011f\u0131n\u0131 ara\u015ft\u0131racakt\u0131r<\/i>
\n<\/strong><\/em><\/h3>\n

Lower Merion, PA - 3 Kas\u0131m 2022 - Nonsense mutasyonlar\u0131 olanlar da dahil olmak \u00fczere 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 vakf\u0131 olan Emily's Entourage (EE), bug\u00fcn Marianne S.'ye $220.000 fon verdi\u011fini duyurdu. Carlon, PhD ve Bel\u00e7ika'daki KU Leuven'den Mattijs Bulcaen, KF'li ki\u015filerde kistik fibroz transmembran reg\u00fclat\u00f6r\u00fc (CFTR) genindeki mutasyonlar\u0131 d\u00fczeltmek i\u00e7in prime editing ad\u0131 verilen yeni bir gen d\u00fczenleme teknolojisinin kullan\u0131m\u0131n\u0131 ara\u015ft\u0131rmak \u00fczere.<\/span><\/p>\n

\"\"

Marianne S. Carlon, PhD ve KU Leuven'den Mattijs Bulcaen, belirli CFTR mutasyonlar\u0131n\u0131 g\u00fcvenli ve verimli bir \u015fekilde d\u00fczeltmek i\u00e7in ana d\u00fczenlemeden yararlanmaya odaklanacak.<\/p><\/div>\n

Prime editing, \u00e7ift sarmall\u0131 DNA k\u0131r\u0131lmalar\u0131 ind\u00fcklenmedi\u011fi i\u00e7in konak DNA'ya zarar vermeden genoma \u00e7ok hassas bir \u015fekilde yeni genetik bilgi yazabilir. Prime editing'in \u00e7ok y\u00f6nl\u00fcl\u00fc\u011f\u00fc, etkinli\u011fi ve g\u00fcvenli\u011fi hakk\u0131nda giderek artan say\u0131da kan\u0131t rapor edilmektedir, ancak KF i\u00e7in potansiyelini ve CFTR mutasyonlar\u0131n\u0131 d\u00fczeltme yetene\u011fini ara\u015ft\u0131rmak i\u00e7in daha fazla ara\u015ft\u0131rmaya ihtiya\u00e7 vard\u0131r.\u00a0<\/span><\/p>\n

Dr. Carlon'un \"\u0130laca diren\u00e7li CFTR mutasyonlar\u0131n\u0131n asal d\u00fczenleme ile yeniden yaz\u0131lmas\u0131\" ba\u015fl\u0131kl\u0131 projesi, belirli CFTR mutasyonlar\u0131n\u0131 d\u00fczeltmek i\u00e7in asal d\u00fczenlemenin kullan\u0131m\u0131n\u0131, asal d\u00fczenlemeyi g\u00fcvenli ve verimli bir \u015fekilde sa\u011flamak i\u00e7in vir\u00fcs benzeri partik\u00fcllerden (VLP'ler) yararlanma olas\u0131l\u0131\u011f\u0131n\u0131 ve VLP'lerin CF mukusundan verimli bir \u015fekilde ge\u00e7ip CF hava yolu epitel h\u00fccrelerine girip gen d\u00fczenlemesi yap\u0131p yapamayaca\u011f\u0131n\u0131 ara\u015ft\u0131r\u0131yor.<\/span><\/p>\n

\"<\/span>Emily's Entourage'\u0131n Ba\u015f Bilimsel Sorumlusu Chandra Ghose, \"EE olarak, KF'li insanlara, \u00f6zellikle de son 10%'de olanlara fayda sa\u011flayabilecek bilimsel at\u0131l\u0131mlar\u0131 h\u0131zland\u0131rma umuduyla, ana d\u00fczenleme gibi y\u00fcksek riskli, y\u00fcksek \u00f6d\u00fcll\u00fc projeleri finanse etmek i\u00e7in her zaman istekli ve heyecanl\u0131y\u0131z\" dedi. \"Bu hibenin finanse edilmesinin \u00f6nemi KF alan\u0131n\u0131n \u00f6tesine uzanmaktad\u0131r ve gen d\u00fczenlemesine uygun di\u011fer genetik hastal\u0131klara da d\u00f6n\u00fc\u015ft\u00fcr\u00fclebilir.\"<\/span><\/p>\n

Bu \u00e7al\u0131\u015fma, CFTR geni i\u00e7indeki mutasyonlar\u0131 hassas bir \u015fekilde d\u00fczeltmek i\u00e7in asal d\u00fczenlemenin yetene\u011fi hakk\u0131nda KF bilimsel toplulu\u011funu bilgilendirecek ve potansiyel bir gen da\u011f\u0131t\u0131m stratejisi olarak VLP'leri daha da ileriye ta\u015f\u0131yacakt\u0131r.<\/span><\/p>\n

Dr. Carlon, \"Bu projenin etkisi, bu hibe kapsam\u0131nda N1303K i\u00e7in birincil d\u00fczenleme stratejisine ili\u015fkin bilgi ve ara\u00e7lar\u0131n geli\u015ftirilmesinde yat\u0131yor, ancak geli\u015ftirilen stratejileri di\u011fer CFTR mutasyonlar\u0131na uyarlama potansiyeli de var\" dedi. \"Gen d\u00fczenlemesi gelecekte ba\u015far\u0131l\u0131 olursa, bu KF'yi iyile\u015ftirme ve b\u00f6ylece KF hastalar\u0131n\u0131n ya\u015fam kalitesini art\u0131rma perspektifi sunar.\"<\/span><\/p>\n

Emily's Entourage'\u0131n finanse etti\u011fi hibeler hakk\u0131nda daha fazla bilgi i\u00e7in <\/span>www.emilysentourage.org\/awarded-grants<\/span><\/a>.\u00a0<\/span><\/p>\n

###<\/span><\/p>\n

Emily'nin \u00c7evresi Hakk\u0131nda<\/b>
\nEmily's Entourage, KF'nin nonsense mutasyonlar\u0131na sahip olanlar da dahil olmak \u00fczere mevcut mutasyon hedefli tedavilerden fayda g\u00f6rmeyen 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, bir KF gen terapisi \u015firketi 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 d\u00fcnya \u00e7ap\u0131nda y\u00fcksek etkili ara\u015ft\u0131rma ve ila\u00e7 geli\u015ftirme \u00e7abalar\u0131na \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>
\nAshling Knight<\/span>
\nPazarlama ve \u0130leti\u015fim Direkt\u00f6r\u00fc<\/span>
\nEmily'nin \u00c7evresi<\/span>
\nashling@emilysentourage.org<\/span><\/a>\u00a0<\/span><\/p>","protected":false},"excerpt":{"rendered":"

Proje, belirli CFTR mutasyonlar\u0131n\u0131 g\u00fcvenli ve etkili bir \u015fekilde d\u00fczeltmek i\u00e7in prime editing ad\u0131 verilen gen d\u00fczenleme teknolojisinden yararlanmaya odaklanacak ve bu d\u00fczenleme kargosunu vir\u00fcs benzeri partik\u00fcller (VLP) arac\u0131l\u0131\u011f\u0131yla iletme olas\u0131l\u0131\u011f\u0131n\u0131 ara\u015ft\u0131racak Lower Merion, PA - 3 Kas\u0131m 2022 - Emily's Entourage (EE), yenilik\u00e7i bir 501(c)3... <\/p>\n

<\/div>\n

Daha fazla bilgi edinin<\/a><\/p>","protected":false},"author":1281,"featured_media":16426,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_seopress_titles_title":"Emily\u2019s Entourage Awards Research Grant to Team Exploring Novel Gene Editing Technology","_seopress_titles_desc":"The project will explore the use of prime editing to correct specific CFTR mutations, the possibility to harness virus-like particles (VLPs) to deliver prime editing in a safe and efficient way, and whether VLPs can efficiently penetrate through CF mucus and enter and gene-edit CF airway epithelial cells.","_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":[178],"tags":[],"class_list":["post-17522","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-press-releases"],"acf":[],"jetpack_featured_media_url":"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/Emilys-Entourage-Logo_Website-Featured-Image.png","jetpack_sharing_enabled":true,"post_mailing_queue_ids":[],"_links":{"self":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/17522","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=17522"}],"version-history":[{"count":5,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/17522\/revisions"}],"predecessor-version":[{"id":20536,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/17522\/revisions\/20536"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media\/16426"}],"wp:attachment":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media?parent=17522"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/categories?post=17522"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/tags?post=17522"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}