{"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\/sv\/440000-awarded-in-round-6-funding-for-cystic-fibrosis-research\/","title":{"rendered":"EE beviljar $440.000 i Round 6-st\u00f6d till forskning om cystisk fibros"},"content":{"rendered":"

Vi \u00e4r mycket glada \u00f6ver att kunna meddela att Emily's Entourage (EE) har beviljat $440.000 i finansiering f\u00f6r tv\u00e5 nya anslag f\u00f6r att fr\u00e4mja forskning och l\u00e4kemedelsutveckling inom cystisk fibros (CF). Anslagen st\u00f6der utvecklingen av en ny nonsensmutationsmodell f\u00f6r illrar och en ny form av genterapi med hj\u00e4lp av lipidnanopartiklar.\u00a0<\/span><\/p>\n

\n

\"Dessa anslag \u00e5terspeglar Emily's Entourages orubbliga engagemang f\u00f6r att fr\u00e4mja livr\u00e4ddande forskning f\u00f6r att fylla kritiska behov och snabbt driva lovande terapeutiska m\u00f6jligheter f\u00f6r CF-gruppen, s\u00e4rskilt de som inte drar nytta av befintliga mutationsinriktade behandlingar.<\/span>\u201c<\/span><\/strong><\/p>\n

- Chandra Ghose, PhD, Chief Scientific Officer, Emily's Entourage<\/strong><\/span><\/p>\n<\/blockquote>\n

MOTTAGARE AV BIDRAG:<\/strong><\/h2>\n
\n

Generering av W1286X cystisk fibros-ferretmodell f\u00f6r unders\u00f6kning av nonsensmutationsriktade terapier
\n<\/b><\/h3>\n

\"HeadshotXingshen Sun, doktorand
\n<\/span>University of Iowa<\/span><\/i><\/p>\n

Innan ett l\u00e4kemedel eller en behandlingsmetod kan anv\u00e4ndas i kliniska pr\u00f6vningar p\u00e5 m\u00e4nniskor m\u00e5ste det f\u00f6rst testas i en djurmodell f\u00f6r att bed\u00f6ma s\u00e4kerhet och effekt. Hittills har det dock inte funnits n\u00e5gra l\u00e4mpliga djurmodeller med en nonsensmutation i cystisk fibros transmembrankonduktansregulator (CFTR). Tidigare studier visar att illern \u00e4r en idealisk art f\u00f6r en CF-modell eftersom den utvecklar en lungsjukdom som leder till andningssvikt och utvecklar pankreassjukdomar som leder till CF-relaterad diabetes. Detta projekt kommer att syfta till att generera en CF-illermodell som inneh\u00e5ller W1286X-mutationen i CFTR-genen, liknande den m\u00e4nskliga W1282X-mutationen. Illermodellen kommer att anv\u00e4ndas f\u00f6r att testa om behandling med flera l\u00e4kemedel kan minska sjukdomssymtomen hos illern. Denna nya W1286X-illermodell ger en ny m\u00f6jlighet att p\u00e5skynda identifieringen av effektiva och s\u00e4kra behandlingsmetoder, vilket avsev\u00e4rt f\u00f6rkortar tiden f\u00f6r att identifiera nya l\u00e4kemedel eller nya behandlingsstrategier f\u00f6r patienter med CFTR-W1282X-mutation, och potentiellt andra nonsensmutationer, p\u00e5 ett s\u00e4tt som cellbaserade system eller gnagarmodeller inte till\u00e5ter.<\/span><\/p>\n

\n

Pulmonell icke-viral leverans av basredigerare riktade mot W1282X
\n<\/b><\/h3>\n

\"HeadshotDebadyuti (Rana) Ghosh, PhD (bilden) och Hugh Smyth, PhD
\n<\/span>University of Texas i Austin<\/span><\/i><\/p>\n

Genf\u00f6r\u00e4ndrande terapi<\/span>som m<\/span>En ny teknik som kan reparera den defekta gen som \u00e4r f\u00f6rknippad med CF har potential att leda till ett permanent botemedel mot sjukdomen. Det finns f\u00f6r n\u00e4rvarande en teknik som effektivt kan korrigera mutationen och \u00e5terst\u00e4lla det nyckelprotein som \u00e4r involverat i normal cell- och lungfunktion. Det \u00e4r dock sv\u00e5rt att leverera denna terapi till patienten utan att den bryts ned i kroppen. Detta projekt kommer att unders\u00f6ka och identifiera farmaceutiska b\u00e4rare - eller material f\u00f6r att leverera genterapilasten - som kan skydda den fr\u00e5n nedbrytning och i slut\u00e4ndan levereras till lungorna.<\/span>gs <\/span>som inhalerade l\u00e4kemedel.<\/span><\/p>\n

 <\/p>\n

 <\/p>\n

\n

Sedan 2011 har EE beviljat mer \u00e4n $5,2 miljoner till 24 forskningsprojekt \u00f6ver hela v\u00e4rlden. Dessa senaste anslag utg\u00f6r den sj\u00e4tte omg\u00e5ngen av anslagsfinansiering som EE beviljar f\u00f6r att p\u00e5skynda utvecklingen av behandlingar f\u00f6r personer med CF som inte har nytta av befintliga mutationsinriktade behandlingar, inklusive personer med nonsensmutationer av CF.\u00a0<\/span><\/p>\n

Vi \u00e4r tacksamma mot anslagsmottagarna och alla engagerade forskare som leder arbetet med att snabbt fylla kritiska behov f\u00f6r alla personer med CF - ingen l\u00e4mnas efter! Se en omfattande lista \u00f6ver alla beviljade forskningsanslag <\/span>h\u00e4r<\/span><\/a>.\u00a0<\/span><\/p>","protected":false},"excerpt":{"rendered":"

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. The grants support the development of a new nonsense mutation ferret model and a novel form of gene therapy using lipid nanoparticles.\u00a0 “These grants reflect Emily’s Entourage’s unwavering… <\/p>\n

<\/div>\n

L\u00e4s mer om<\/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\/sv\/wp-json\/wp\/v2\/posts\/16623","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=16623"}],"version-history":[{"count":5,"href":"https:\/\/www.emilysentourage.org\/sv\/wp-json\/wp\/v2\/posts\/16623\/revisions"}],"predecessor-version":[{"id":16629,"href":"https:\/\/www.emilysentourage.org\/sv\/wp-json\/wp\/v2\/posts\/16623\/revisions\/16629"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/sv\/wp-json\/wp\/v2\/media\/16748"}],"wp:attachment":[{"href":"https:\/\/www.emilysentourage.org\/sv\/wp-json\/wp\/v2\/media?parent=16623"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/sv\/wp-json\/wp\/v2\/categories?post=16623"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/sv\/wp-json\/wp\/v2\/tags?post=16623"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}