{"id":22179,"date":"2023-12-16T21:12:50","date_gmt":"2023-12-16T21:12:50","guid":{"rendered":"https:\/\/dupmecp2.eu\/huidagene-eine-vielversprechende-crispr-cas13-technologie-aus-shanghai-china\/"},"modified":"2023-12-20T18:46:19","modified_gmt":"2023-12-20T18:46:19","slug":"huidagene-eine-vielversprechende-crispr-cas13-technologie-aus-shanghai-china","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/el\/huidagene-eine-vielversprechende-crispr-cas13-technologie-aus-shanghai-china\/","title":{"rendered":"Huidagene: \u039c\u03b9\u03b1 \u03c0\u03bf\u03bb\u03cd \u03c3\u03b7\u03bc\u03b1\u03bd\u03c4\u03b9\u03ba\u03ae \u03c4\u03b5\u03c7\u03bd\u03bf\u03bb\u03bf\u03b3\u03af\u03b1 CRISPR-CAS13 \u03b1\u03c0\u03cc \u03c4\u03b7 \u03a3\u03b1\u03b3\u03ba\u03ac\u03b7 (\u039a\u03af\u03bd\u03b1)"},"content":{"rendered":"

\u0397 Huidagene Therapeutics \u03b5\u03af\u03bd\u03b1\u03b9 \u03bc\u03b9\u03b1 \u03b5\u03c4\u03b1\u03b9\u03c1\u03b5\u03af\u03b1 \u03b2\u03b9\u03bf\u03c4\u03b5\u03c7\u03bd\u03bf\u03bb\u03bf\u03b3\u03af\u03b1\u03c2 \u03bc\u03b5 \u03ad\u03b4\u03c1\u03b1 \u03c4\u03b7 \u03a3\u03b1\u03b3\u03ba\u03ac\u03b7 \u03ba\u03b1\u03b9 \u03c4\u03bf \u039d\u03b9\u03bf\u03c5 \u03a4\u03b6\u03ad\u03c1\u03c3\u03b5\u03ca, \u03b7 \u03bf\u03c0\u03bf\u03af\u03b1 \u03b5\u03c0\u03b9\u03ba\u03b5\u03bd\u03c4\u03c1\u03ce\u03bd\u03b5\u03c4\u03b1\u03b9 \u03c3\u03c4\u03b7\u03bd \u03b1\u03bd\u03b1\u03ba\u03ac\u03bb\u03c5\u03c8\u03b7, \u03c4\u03b7 \u03bc\u03b7\u03c7\u03b1\u03bd\u03b9\u03ba\u03ae \u03ba\u03b1\u03b9 \u03c4\u03b7\u03bd \u03b1\u03bd\u03ac\u03c0\u03c4\u03c5\u03be\u03b7 \u03bd\u03ad\u03c9\u03bd \u03c6\u03b1\u03c1\u03bc\u03ac\u03ba\u03c9\u03bd \u03b2\u03b1\u03c3\u03b9\u03c3\u03bc\u03ad\u03bd\u03c9\u03bd \u03c3\u03c4\u03bf CRISPR. \u0397 \u03b5\u03c4\u03b1\u03b9\u03c1\u03b5\u03af\u03b1 \u03b5\u03c0\u03b9\u03ba\u03b5\u03bd\u03c4\u03c1\u03ce\u03bd\u03b5\u03c4\u03b1\u03b9 \u03c3\u03c4\u03b9\u03c2 \u03bc\u03c5\u03ca\u03ba\u03ad\u03c2, \u03bf\u03c6\u03b8\u03b1\u03bb\u03bc\u03b9\u03ba\u03ad\u03c2 \u03ba\u03b1\u03b9 \u03bd\u03b5\u03c5\u03c1\u03bf\u03bb\u03bf\u03b3\u03b9\u03ba\u03ad\u03c2 \u03b4\u03b9\u03b1\u03c4\u03b1\u03c1\u03b1\u03c7\u03ad\u03c2.<\/p>\n\n\n\n

\u0397 Huidagene \u03ad\u03c7\u03b5\u03b9 \u03b1\u03bd\u03b1\u03c0\u03c4\u03cd\u03be\u03b5\u03b9 \u03c4\u03bf HG204, \u03ad\u03bd\u03b1\u03bd \u03b1\u03b4\u03b5\u03bd\u03bf-\u03b1\u03c3\u03bf\u03b6\u03b9\u03c4\u03ad\u03bd\u03b9\u03bf \u03b9\u03bf\u03b3\u03b5\u03bd\u03ae (AAV) Vektor, \u03bf \u03bf\u03c0\u03bf\u03af\u03bf\u03c2 \u03c6\u03ad\u03c1\u03bd\u03b5\u03b9 \u03ad\u03bd\u03b1 \u03bd\u03ad\u03bf \u03c3\u03cd\u03bc\u03c0\u03bb\u03b5\u03b3\u03bc\u03b1 CRISPR-Cas13-Komplex in Zellen. HG204 wurde f\u00fcr das MECP2-Duplikationssyndrom entwickelt und zielt darauf ab, die \u00dcberproduktion des MeCP2-Proteins zu regulieren, indem es den Abbau der \u00fcberexprimierten MECP2-RNA induziert. \u0391\u03c5\u03c4\u03cc \u03c4\u03bf \u03ba\u03b1\u03b9\u03bd\u03bf\u03c4\u03cc\u03bc\u03bf \u03c5\u03c0\u03bf\u03c8\u03ae\u03c6\u03b9\u03bf \u03c6\u03ac\u03c1\u03bc\u03b1\u03ba\u03bf \u03ad\u03c7\u03b5\u03b9 \u03c4\u03b7 \u03b4\u03c5\u03bd\u03b1\u03c4\u03cc\u03c4\u03b7\u03c4\u03b1 \u03bd\u03b1 \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03cd\u03c3\u03b5\u03b9 \u03c4\u03bf MDS \u03bc\u03ad\u03c3\u03c9 \u03bc\u03b9\u03b1\u03c2 \u03bc\u03cc\u03bd\u03bf \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03af\u03b1\u03c2.<\/strong><\/p>\n\n\n\n

<\/div>\n\n\n\n

\u0397 \u03bf\u03c1\u03b3\u03ac\u03bd\u03c9\u03c3\u03ae \u03bc\u03b1\u03c2 \u03ad\u03c7\u03b5\u03b9 \u03c0\u03b1\u03c1\u03b1\u03b3\u03c9\u03b3\u03b9\u03ba\u03ad\u03c2 \u03c3\u03c5\u03bd\u03bf\u03bc\u03b9\u03bb\u03af\u03b5\u03c2 \u03bc\u03b5 \u03c4\u03bf\u03c5\u03c2 F\u00fchrungskr\u00e4ften \u03c4\u03b7\u03c2 Huidagene gef\u00fchrt, um mehr \u00fcber diese innovative Technologie und ihre Bedeutung f\u00fcr die Zukunft zu erfahren. Pr\u00e4klinische Studien mit HG204 an einem humanisierten MDS-Mausmodell zeigten eine signifikante Reduktion des MeCP2-Proteins und eine Umkehrung der Symptome.<\/strong> Diese Ergebnisse sind ermutigend, da es auch um den zweiten Nachweis der Umkehrbarkeit von MDS-Symptomen nach der Regulierung der MECP2-Proteinspiegel handelt. (wie zuvor durch die Verabreichung des ASO durch Prof. H. Zoghbi & Team nachgewiesen).<\/p>\n\n\n\n

<\/div>\n\n\n\n

\u0397 Huidagene schlie\u00dft derzeit die pr\u00e4klinischen Studien zu HG204 ab und bereitet sich auf eine klinische Studie f\u00fcr Patienten in China vor, die im Sommer 2024 beginnen soll.<\/strong> Sollte diese Studie erfolgreich verlaufen, plant Huidagene, in den Jahren 2025-2026 klinische Studien in anderen L\u00e4ndern, wie den USA und Europa, durchzuf\u00fchren.<\/p>\n\n\n\n


Am 31. Oktober hat die FDA (Food and Drug Administration, die Gesundheitsbeh\u00f6rde der USA) sowohl die Auszeichnung als seltene p\u00e4diatrische Erkrankung als auch den Orphan-Drug-Status f\u00fcr HG204 verliehen. Diese beh\u00f6rdliche Festlegung ist ein erster Schritt auf dem Weg zu einer klinischen Studie au\u00dferhalb Chinas und soll eine beschleunigte Pr\u00fcfung des Dossiers bei der Registrierung erm\u00f6glichen. Obwohl die Nachricht v\u00f6llig unerwartet war, bringt sie der Gemeinschaft zus\u00e4tzliche Hoffnung.<\/p>\n\n\n\n

Unser Team wird mit Huidagene in Verbindung bleiben und unsere Gemeinschaft \u00fcber alle neuen developments auf dem Laufenden halten.<\/p>\n\n\n\n

\"\"<\/figure>\n\n\n\n
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Zur Pressemitteilung<\/a><\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"

Huidagene Therapeutics ist ein Biotechnologieunternehmen mit Sitz in Shanghai und New Jersey, das sich auf die Entdeckung, das Engineering und die Entwicklung neuartiger CRISPR-basierter Medikamente konzentriert. Das Unternehmen konzentriert sich […]<\/p>","protected":false},"author":1,"featured_media":22152,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[57],"tags":[],"class_list":["post-22179","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-aktualitat"],"yoast_head":"\nHuidagene: Eine vielversprechende CRISPR-CAS13 Technologie aus Shanghai (China) - DupMECP2<\/title>\n<meta name=\"description\" content=\"Eine m\u00f6gliche neue Perspektive f\u00fcr die Behandlung des MECP2-Duplikationssyndroms mit CRIPSR-Cas13 von Huidagene. 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