{"id":1404,"date":"2023-12-16T17:30:00","date_gmt":"2023-12-16T16:30:00","guid":{"rendered":"https:\/\/remeds.org\/?p=1404"},"modified":"2025-02-06T18:04:25","modified_gmt":"2025-02-06T17:04:25","slug":"huidagene-a-promising-crispr-cas13-technology-from-shanghai-china","status":"publish","type":"post","link":"https:\/\/remeds.org\/hu\/2023\/12\/16\/huidagene-a-promising-crispr-cas13-technology-from-shanghai-china\/","title":{"rendered":"Huidagene: CRISPR-CAS13 technol\u00f3gia Sanghajb\u00f3l (K\u00edna)"},"content":{"rendered":"

A Huidagene Therapeutics egy Sanghajban \u00e9s New Jerseyben m\u0171k\u00f6d\u0151 biotechnol\u00f3giai v\u00e1llalat, amely \u00faj CRISPR-alap\u00fa gy\u00f3gyszerek felfedez\u00e9s\u00e9re, tervez\u00e9s\u00e9re \u00e9s fejleszt\u00e9s\u00e9re \u00f6sszpontos\u00edt. A v\u00e1llalat izom-, szem\u00e9szeti \u00e9s neurol\u00f3giai rendelleness\u00e9gekre \u00f6sszpontos\u00edt.<\/p>\n\n\n\n

A Huidagene kifejlesztette a HG204-et, egy adeno-asszoci\u00e1lt v\u00edrusvektort (AAV), amely egy \u00faj CRISPR-Cas13 komplexet juttat a sejtekbe. A MECP2 duplik\u00e1ci\u00f3s szindr\u00f3m\u00e1ra tervezt\u00e9k, A HG204 c\u00e9lja a MeCP2 feh\u00e9rjeszint t\u00faltermel\u00e9s\u00e9nek szab\u00e1lyoz\u00e1sa az\u00e1ltal, hogy a t\u00falexpressz\u00e1lt MECP2 RNS degrad\u00e1ci\u00f3j\u00e1t induk\u00e1lja.<\/strong> Ez az innovat\u00edv gy\u00f3gyszerjel\u00f6lt mag\u00e1ban hordozza az MDS gy\u00f3gy\u00edt\u00e1s\u00e1nak lehet\u0151s\u00e9g\u00e9t egyetlen bead\u00e1ssal.<\/p>\n\n\n\n

Szervezet\u00fcnk term\u00e9keny megbesz\u00e9l\u00e9seket folytatott a Huidagene vezet\u0151ivel, hogy t\u00f6bbet tudjunk meg err\u0151l az innovat\u00edv technol\u00f3gi\u00e1r\u00f3l \u00e9s arr\u00f3l, hogy mit jelent a j\u00f6v\u0151re n\u00e9zve. A HG204 haszn\u00e1lat\u00e1val v\u00e9gzett preklinikai vizsg\u00e1latok egy humaniz\u00e1lt MDS eg\u00e9rmodellben a MeCP2 feh\u00e9rje jelent\u0151s cs\u00f6kken\u00e9s\u00e9t \u00e9s a t\u00fcnetek visszaford\u00edt\u00e1s\u00e1t mutatt\u00e1k.<\/strong> B\u00e1r ezek b\u00edztat\u00f3 eredm\u00e9nyek, ez a m\u00e1sodik bizony\u00edt\u00e9k az MDS t\u00fcneteinek visszaford\u00edthat\u00f3s\u00e1g\u00e1ra a MECP2 feh\u00e9rjeszint szab\u00e1lyoz\u00e1s\u00e1t k\u00f6vet\u0151en (amint azt kor\u00e1bban Prof. H. Zoghbi \u00e9s csapata az ASO bead\u00e1s\u00e1val bizony\u00edtotta).<\/p>\n\n\n\n

A Huidagene jelenleg a HG204-gyel v\u00e9gzett preklinikai vizsg\u00e1latok befejez\u00e9s\u00e9n dolgozik, \u00e9s felk\u00e9sz\u00fcl a k\u00ednai betegek klinikai vizsg\u00e1lat\u00e1ra, amely a tervek szerint 2024 k\u00f6zep\u00e9n kezd\u0151dik. <\/strong>Ha ez a vizsg\u00e1lat sikeres lesz, a Huidagene 2025-2026-ban m\u00e1s orsz\u00e1gokban, p\u00e9ld\u00e1ul az Egyes\u00fclt \u00c1llamokban \u00e9s Eur\u00f3p\u00e1ban is tervez klinikai vizsg\u00e1latokat.<\/p>\n\n\n\n


Okt\u00f3ber 31-\u00e9n az FDA (Food and Drug Administration, az Egyes\u00fclt \u00c1llamok eg\u00e9szs\u00e9g\u00fcgyi hat\u00f3s\u00e1ga) megadta a HG204-nek a ritka gyermekbetegs\u00e9g \u00e9s a ritka betegs\u00e9gek gy\u00f3gyszere min\u0151s\u00edt\u00e9st. Ez a hat\u00f3s\u00e1gi megjel\u00f6l\u00e9s az els\u0151 l\u00e9p\u00e9s a K\u00edn\u00e1n k\u00edv\u00fcli klinikai vizsg\u00e1lat fel\u00e9, \u00e9s lehet\u0151v\u00e9 teszi a dosszi\u00e9 gyors\u00edtott fel\u00fclvizsg\u00e1lat\u00e1t a gy\u00f3gyszer bejegyz\u00e9se ut\u00e1n. B\u00e1r a h\u00edr teljesen v\u00e1ratlan volt, tov\u00e1bbi rem\u00e9nyt ad a k\u00f6z\u00f6ss\u00e9gnek.<\/p>\n\n\n\n

Csapatunk kapcsolatban marad a Huidagene-nel, \u00e9s folyamatosan t\u00e1j\u00e9koztatja k\u00f6z\u00f6ss\u00e9g\u00fcnket az \u00faj fejlem\u00e9nyekr\u0151l.<\/p>\n\n\n\n

\"\"<\/figure>\n\n\n\n

A sajt\u00f3k\u00f6zlem\u00e9nyhez<\/a><\/p>","protected":false},"excerpt":{"rendered":"

Huidagene Therapeutics is a biotechnology company based in Shanghai and New Jersey, focusing on discovering, engineering, and developing novel CRISPR-based medicines. The company focuses on muscular, ophthalmic and neurological disorders. Huidagene has developed HG204, an adeno-associated viral (AAV) vector delivering a novel CRISPR-Cas13 complex into cells. Designed for MECP2 duplication syndrome, HG204 aims to regulate the […]<\/p>\n","protected":false},"author":73,"featured_media":1429,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_jf_save_progress":"","footnotes":""},"categories":[12,10,9],"tags":[],"class_list":["post-1404","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-crispr","category-cure","category-research"],"_links":{"self":[{"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/posts\/1404","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/users\/73"}],"replies":[{"embeddable":true,"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/comments?post=1404"}],"version-history":[{"count":1,"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/posts\/1404\/revisions"}],"predecessor-version":[{"id":1405,"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/posts\/1404\/revisions\/1405"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/media\/1429"}],"wp:attachment":[{"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/media?parent=1404"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/categories?post=1404"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/tags?post=1404"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}