{"id":17502,"date":"2022-11-02T07:38:39","date_gmt":"2022-11-02T11:38:39","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?page_id=17502"},"modified":"2023-12-20T05:15:53","modified_gmt":"2023-12-20T10:15:53","slug":"emilys-entourage-awards-220000-to-university-of-iowa-laboratory-to-advance-research-into-correcting-nonsense-mutation-cftr-function","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/sv\/emilys-entourage-awards-220000-to-university-of-iowa-laboratory-to-advance-research-into-correcting-nonsense-mutation-cftr-function\/","title":{"rendered":"Emily's Entourage beviljar $220.000 till University of Iowa Laboratory f\u00f6r att fr\u00e4mja forskning om korrigering av nonsensmutationen CFTR:s funktion"},"content":{"rendered":"

Projektet introducerar nya system f\u00f6r att korrigera CFTR-proteinet (cystic fibrosis transmembrane conductance regulator) i luftv\u00e4garna hos personer med nonsensmutationer av CF<\/i>
\n<\/strong><\/em><\/h3>\n

2 november 2022 - Emily's Entourage (EE), en innovativ 501(c)3-stiftelse som p\u00e5skyndar forskning f\u00f6r nya behandlingar och ett botemedel f\u00f6r de sista 10% av personer med cystisk fibros (CF), inklusive de med nonsensmutationer, meddelade idag att de har beviljat $220.000 i finansiering till Patrick L. Sinn, PhD, fr\u00e5n University of Iowa f\u00f6r att st\u00f6dja hans forskning om korrigering av CFTR-proteinet hos personer med minst en nonsensmutation av CF.<\/span><\/p>\n

\"\"

Patrick L. Sinn, PhD, vid University of Iowa, driver forskning som syftar till att \u00e5stadkomma platsspecifik reparation av CFTR-mutationer med hj\u00e4lp av ett konstruerat enzym, en s\u00e5 kallad Adenine Base Editor (ABE).<\/p><\/div>\n

Kunskapen om CFTR:s funktion och uttryck i olika celltyper har utvecklats mycket sedan CFTR uppt\u00e4cktes 1989. F\u00f6r n\u00e4rvarande har 90% av alla personer med CF nytta av mutationsinriktade behandlingar som \u00e5terst\u00e4ller funktionen hos deras CFTR-mutationer. Men de \u00e5terst\u00e5ende 10% av personer med CF kan inte dra nytta av dessa l\u00e4kemedel eftersom de producerar f\u00f6r lite eller inget funktionellt protein.<\/span><\/p>\n

Projektet, som har titeln \"Correction of nonsense CFTR mutations using extracellular vesicle-delivered base editors\", syftar till att \u00e5stadkomma platsspecifik reparation av CFTR-mutationer med hj\u00e4lp av ett konstruerat enzym, en s\u00e5 kallad Adenine Base Editor (ABE). Dr. Sinns team kommer att fokusera p\u00e5 att leverera ABE till tillr\u00e4ckligt m\u00e5nga av de l\u00e4mpliga luftv\u00e4gscellerna f\u00f6r att vara terapeutiskt.<\/span><\/p>\n

\"Dr. Sinns laboratorium har en dokumenterad erfarenhet av att anv\u00e4nda banbrytande teknik f\u00f6r genredigering\", s\u00e4ger Chandra Ghose, PhD, Chief Scientific Officer p\u00e5 Emily's Entourage. \"Vi f\u00f6rv\u00e4ntar oss att enorma framsteg kommer att g\u00f6ras inom CF-omr\u00e5det och kommer att ge ytterligare information om utvecklingen av nya terapier, med s\u00e4rskild uppm\u00e4rksamhet p\u00e5 den slutliga 10% hos personer med CF som har s\u00e4llsynta och nonsensmutationer<\/span>.\"<\/span><\/p>\n

Projektet kommer att anv\u00e4nda naturligt f\u00f6rekommande partiklar, s\u00e5 kallade extracellul\u00e4ra vesiklar (EVs), som de flesta celler i kroppen producerar. Dessa partiklar \u00e4r mycket sm\u00e5 och har utvecklats till att vara cellul\u00e4ra budb\u00e4rare som kan leverera sm\u00e5 molekyler och proteiner mellan cellerna. Dr. Sinns laboratorium har tidigare visat att EVs kan leverera sm\u00e5 RNA och proteiner till luftv\u00e4gsceller. Som en del av detta anslag kommer de att testa om de kan paketera och leverera ABEs f\u00f6r att korrigera m\u00e5nga av de s\u00e4llsynta nonsensmutationerna i CFTR.<\/span><\/p>\n

\"Jag \u00e4r mycket entusiastisk \u00f6ver potentialen i denna metod\", s\u00e4ger Dr Sinn, professor vid Department of Pediatrics, Division of Pulmonary Medicine vid University of Iowa. \"Om v\u00e5ra experiment blir framg\u00e5ngsrika kommer vi att ha en snabb och billig teknik f\u00f6r att utf\u00f6ra genredigeringsexperiment i ett kraftfullt modellsystem och en potentiell behandling f\u00f6r genetiska sjukdomar som cystisk fibros. V\u00e5rt slutgiltiga m\u00e5l \u00e4r att uppn\u00e5 livsl\u00e5ng CFTR-genkorrigering genom en enda dos av denna aerosolbehandling.\"<\/span><\/p>\n

L\u00e4s mer om de bidrag som Emily's Entourage har finansierat p\u00e5 <\/span>www.emilysentourage.org\/awarded-grants<\/span><\/a>.\u00a0<\/span><\/p>\n

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

Om Emily's Entourage<\/b><\/p>\n

Emily's Entourage \u00e4r en innovativ 501(c)3 som p\u00e5skyndar forskning f\u00f6r nya behandlingar och ett botemedel f\u00f6r individer i den sista 10% av cystisk fibros (CF)-populationen som inte drar nytta av de mutationsinriktade terapier som f\u00f6r n\u00e4rvarande finns tillg\u00e4ngliga, inklusive de med nonsensmutationer av CF. Sedan 2011 har Emily's Entourage beviljat miljontals dollar i forskningsanslag, lanserat ett genterapibolag f\u00f6r CF, utvecklat ett patientregister och ett matchningsprogram f\u00f6r kliniska pr\u00f6vningar f\u00f6r att p\u00e5skynda rekryteringen till kliniska pr\u00f6vningar samt lett v\u00e4rldsomsp\u00e4nnande insatser f\u00f6r att driva forskning och l\u00e4kemedelsutveckling med stor genomslagskraft. Organisationen har omn\u00e4mnts i nationella medier, bland annat New York Times, STAT, CNN, People med flera. L\u00e4s mer p\u00e5 <\/span>emilysentourage.org<\/span><\/a>.\u00a0<\/span><\/p>\n

Kontakt med media<\/b>
\nAshling Knight<\/span>
\nDirekt\u00f6r f\u00f6r marknadsf\u00f6ring och kommunikation<\/span>
\nEmily's Entourage<\/span>
\nashling@emilysentourage.org<\/span><\/a>\u00a0<\/span><\/p>","protected":false},"excerpt":{"rendered":"

The project introduces novel systems for correcting the cystic fibrosis transmembrane conductance regulator (CFTR) protein in the airways of people with nonsense mutations of CF Nov. 2, 2022 \u2014 Emily\u2019s Entourage (EE), an innovative 501(c)3 foundation that accelerates research for new treatments and a cure for the final 10% of people with cystic fibrosis (CF),… <\/p>\n

<\/div>\n

L\u00e4s mer om<\/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 $220,000 to University of Iowa Laboratory to Advance Research Into Correcting Nonsense Mutation CFTR Function","_seopress_titles_desc":"The project introduces novel systems for correcting the cystic fibrosis transmembrane conductance regulator (CFTR) protein in the airways of people with nonsense mutations of CF.","_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-17502","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\/sv\/wp-json\/wp\/v2\/posts\/17502","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.emilysentourage.org\/sv\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.emilysentourage.org\/sv\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/sv\/wp-json\/wp\/v2\/users\/1281"}],"replies":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/sv\/wp-json\/wp\/v2\/comments?post=17502"}],"version-history":[{"count":5,"href":"https:\/\/www.emilysentourage.org\/sv\/wp-json\/wp\/v2\/posts\/17502\/revisions"}],"predecessor-version":[{"id":20533,"href":"https:\/\/www.emilysentourage.org\/sv\/wp-json\/wp\/v2\/posts\/17502\/revisions\/20533"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/sv\/wp-json\/wp\/v2\/media\/16426"}],"wp:attachment":[{"href":"https:\/\/www.emilysentourage.org\/sv\/wp-json\/wp\/v2\/media?parent=17502"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/sv\/wp-json\/wp\/v2\/categories?post=17502"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/sv\/wp-json\/wp\/v2\/tags?post=17502"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}