{"id":22829,"date":"2023-12-16T21:12:50","date_gmt":"2023-12-16T21:12:50","guid":{"rendered":"https:\/\/dupmecp2.eu\/huidagene-una-promettente-tecnologia-crispr-cas13-da-shanghai-cina\/"},"modified":"2024-01-31T12:43:10","modified_gmt":"2024-01-31T12:43:10","slug":"huidagene-obiecujaca-technologia-crispr-cas13-z-szanghaju-w-chinach","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/pl\/huidagene-obiecujaca-technologia-crispr-cas13-z-szanghaju-w-chinach\/","title":{"rendered":"Huidagene: obiecuj\u0105ca technologia CRISPR-CAS13 z Szanghaju (Chiny)"},"content":{"rendered":"<p>Huidagene Therapeutics to firma biotechnologiczna z siedzib\u0105 w Shangai i New Jersey, zajmuj\u0105ca si\u0119 poszukiwaniem i rozwojem nowych lek\u00f3w opartych na technologii CRISPR. Firma koncentruje si\u0119 na chorobach uk\u0142adu mi\u0119\u015bniowo-szkieletowego i neurologicznych.<\/p>\n\n<p>Firma Huidagene opracowa\u0142a HG204, wektor wirusowy powi\u0105zany z adenowirusem (AAV), kt\u00f3ry przenosi innowacyjny kompleks CRISPR-Cas13 do kom\u00f3rek. Appositamente progettato per la sindrome di duplicazione del gene MECP2, <strong>HG204 reguluje produkcj\u0119 bia\u0142ka MeCP2, indukuj\u0105c degradacj\u0119 RNA MECP2.<\/strong> Ten innowacyjny kandydat na lek farmakologiczny ma potencja\u0142 do leczenia zespo\u0142u duplikacji genu MECP2 za pomoc\u0105 pojedynczej syntezy.<\/p>\n\n<div style=\"height:25px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n<p>Nasza organizacja przeprowadzi\u0142a owocne rozmowy z dyrektorami Huidagene, aby pog\u0142\u0119bi\u0107 nasze zrozumienie tej innowacyjnej technologii i dowiedzie\u0107 si\u0119, co oznacza ona dla przysz\u0142o\u015bci. <strong>Badania przedkliniczne, w kt\u00f3rych wykorzystano HG204 w modelu topo umanizzato, wykaza\u0142y znaczn\u0105 redukcj\u0119 bia\u0142ka MeCP2 i inwersj\u0119 sintomi.<\/strong> Opr\u00f3cz tego, \u017ce wyniki te s\u0105 niepoprawne, przedstawiaj\u0105 one r\u00f3wnie\u017c drug\u0105 dow\u00f3d na odwracalno\u015b\u0107 objaw\u00f3w syndromu, je\u015bli interweniuje si\u0119 w celu regulacji poziomu bia\u0142ka MECP2 (co zosta\u0142o wcze\u015bniej potwierdzone przez prof. H. Zoghbi i jego zesp\u00f3\u0142 w trakcie badania ASO).<\/p>\n\n<div style=\"height:25px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n<p><strong>Huidagene ko\u0144czy obecnie badania przedkliniczne na HG204 i przygotowuje si\u0119 do badania klinicznego na pacjentach w Chinach, kt\u00f3re mo\u017ce rozpocz\u0105\u0107 si\u0119 w 2024 roku. <\/strong>Je\u015bli wyniki tego badania oka\u017c\u0105 si\u0119 pozytywne, Huidagene planuje przeprowadzenie test\u00f3w klinicznych w innych krajach, takich jak Stany Zjednoczone i Europa, w latach 2025-2026.<\/p>\n\n<p><br\/>W dniu 31 pa\u017adziernika FDA (Food and Drug Administration, organ ds. zdrowia w Stanach Zjednoczonych) przyzna\u0142 HG204 oznaczenie sia di farmaco per malattia pediatrica rara che di farmaco orfano. To oznaczenie regulacyjne stanowi pierwszy krok w kierunku bada\u0144 klinicznych poza Chinami i pozwoli na przyspieszon\u0105 rewizj\u0119 dokumentacji podczas procesu rejestracji leku. Nawet je\u015bli powiadomienie pozostanie bez odzewu, da to spo\u0142eczno\u015bci kolejny pow\u00f3d do nadziei.<\/p>\n\n<p>Nasz zesp\u00f3\u0142 pozostaje w kontakcie z Huidagene i informuje nasz\u0105 spo\u0142eczno\u015b\u0107 o wszelkich nowych rozwi\u0105zaniach.<\/p>\n\n<figure class=\"wp-block-image size-large is-resized\"><img decoding=\"async\" data-src=\"https:\/\/dupmecp2.eu\/wp-content\/uploads\/2023\/12\/Huidagene_Pipeline_2023_white-edited.png\" alt=\"\" class=\"wp-image-22144 lazyload\" style=\"--smush-placeholder-width: 1920px; --smush-placeholder-aspect-ratio: 1920\/1080;width:840px;height:auto\" src=\"data:image\/svg+xml;base64,PHN2ZyB3aWR0aD0iMSIgaGVpZ2h0PSIxIiB4bWxucz0iaHR0cDovL3d3dy53My5vcmcvMjAwMC9zdmciPjwvc3ZnPg==\" \/><\/figure>\n\n<div class=\"wp-block-buttons is-layout-flex wp-block-buttons-is-layout-flex\">\n<div class=\"wp-block-button\"><a class=\"wp-block-button__link has-background wp-element-button\" href=\"https:\/\/www.huidagene.com\/new\/news\/48\" style=\"border-radius:16px;background-color:#f7a13c\" target=\"_blank\" rel=\"noreferrer noopener\">Vai al comunicato stampa<\/a><\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"<p>Huidagene Therapeutics \u00e8 un&rsquo;azienda biotecnologica basata a Shangai e New Jersey, attiva nella scoperta e sviluppo di nuove medicine basate sulla tecnologia CRISPR. L&rsquo;azienda si focalizza su malattie muscolari, oftalmiche [&hellip;]<\/p>","protected":false},"author":1,"featured_media":22153,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[137],"tags":[],"class_list":["post-22829","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-notizie"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v25.8 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>Huidagene: una promettente tecnologia CRISPR-CAS13 da Shanghai (Cina) - DupMECP2<\/title>\n<meta name=\"description\" content=\"Una nuova potenziale prospettiva per il trattamento della Sindrome da Duplicazione di MECP2 con il sistema CRISPR-Cas13 di Huidagene. Il candidato farmaco HG204 ha dimostrato una significativa riduzione della proteina MeCP2 e un&#039;inversione dei sintomi.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/dupmecp2.eu\/pl\/huidagene-obiecujaca-technologia-crispr-cas13-z-szanghaju-w-chinach\/?lang=it\" \/>\n<meta property=\"og:locale\" content=\"pl_PL\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Huidagene: una promettente tecnologia CRISPR-CAS13 da Shanghai (Cina) - DupMECP2\" \/>\n<meta property=\"og:description\" content=\"Una nuova potenziale prospettiva per il trattamento della Sindrome da Duplicazione di MECP2 con il sistema CRISPR-Cas13 di Huidagene. 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