{"id":3562,"date":"2025-06-02T08:54:11","date_gmt":"2025-06-02T06:54:11","guid":{"rendered":"https:\/\/remeds.org\/?p=3562"},"modified":"2025-11-14T09:26:07","modified_gmt":"2025-11-14T08:26:07","slug":"umass-licenses-sirna-mecp2-duplication-syndrome","status":"publish","type":"post","link":"https:\/\/remeds.org\/pl\/2025\/06\/02\/umass-licenses-sirna-mecp2-duplication-syndrome\/","title":{"rendered":"Nowy krok naprz\u00f3d w poszukiwaniu lekarstwa: UMass Chan licencjonuje technologi\u0119 siRNA \u00a0"},"content":{"rendered":"<p class=\"wp-block-paragraph\">UMass Chan Medical School udzieli\u0142a licencji na obiecuj\u0105c\u0105 technologi\u0119 opart\u0105 na siRNA firmie M2DS, startupowi zajmuj\u0105cemu si\u0119 rozwijaniem tej technologii jako leczenia zespo\u0142u duplikacji MECP2. Jest to znacz\u0105cy krok naprz\u00f3d w prze\u0142o\u017ceniu najnowszych bada\u0144 na realia kliniczne.<a href=\"https:\/\/dupmecp2.eu\/recruitement-hero\/\"><\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Technologia zosta\u0142a opracowana w laboratorium dr Khvorovej, a projekt by\u0142 prowadzony przez Vignesha Hariharana. Wczesne badania by\u0142y mo\u017cliwe dzi\u0119ki finansowaniu od Cure MDS i Rett Syndrome Research Trust. Ten siRNA dzia\u0142a poprzez celowanie i degradacj\u0119 informacyjnego RNA odpowiedzialnego za nadprodukcj\u0119 bia\u0142ka MeCP2. <\/p>\n\n\n\n<p class=\"wp-block-paragraph\">W badaniach na myszach dawka skutecznie dotar\u0142a do m\u00f3zgu i rdzenia kr\u0119gowego i mo\u017ce wymaga\u0107 podawania tylko raz lub dwa razy w roku. Poprzez licencjonowanie tej technologii firmie M2DS, terapia wchodzi teraz na \u015bcie\u017ck\u0119, gdzie mo\u017ce uzyska\u0107 wsparcie potrzebne do przej\u015bcia do bada\u0144 klinicznych na ludziach. <\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Opr\u00f3cz trwaj\u0105cych bada\u0144 klinicznych prowadzonych przez Ionis i HuidaGene, osi\u0105gni\u0119cie to mo\u017ce stanowi\u0107 obiecuj\u0105ce uzupe\u0142nienie portfolio potencjalnych terapii tej rzadkiej i z\u0142o\u017conej choroby.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><a href=\"https:\/\/bridge.umassmed.edu\/s\/news\/news-article\/umass-chan-licenses-sirna-therapeutics-for-mecp2-duplication-syndrome-MCMWKNCJYQ25CQHF5YITTOM757HU\">Zobacz komunikat prasowy<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>UMass Chan Medical School has licensed a promising siRNA-based technology to M2DS, a startup company dedicated to advancing the technology as a treatment for MECP2 Duplication Syndrome. This marks a major step forward in translating cutting-edge research into clinical reality. The technology was developed in the lab of Dr. Khvorova, with the project led by [&hellip;]<\/p>\n","protected":false},"author":114,"featured_media":3563,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_jf_save_progress":"","footnotes":""},"categories":[9,24],"tags":[],"class_list":["post-3562","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-research","category-sirna"],"_links":{"self":[{"href":"https:\/\/remeds.org\/pl\/wp-json\/wp\/v2\/posts\/3562","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/remeds.org\/pl\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/remeds.org\/pl\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/remeds.org\/pl\/wp-json\/wp\/v2\/users\/114"}],"replies":[{"embeddable":true,"href":"https:\/\/remeds.org\/pl\/wp-json\/wp\/v2\/comments?post=3562"}],"version-history":[{"count":3,"href":"https:\/\/remeds.org\/pl\/wp-json\/wp\/v2\/posts\/3562\/revisions"}],"predecessor-version":[{"id":3567,"href":"https:\/\/remeds.org\/pl\/wp-json\/wp\/v2\/posts\/3562\/revisions\/3567"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/remeds.org\/pl\/wp-json\/wp\/v2\/media\/3563"}],"wp:attachment":[{"href":"https:\/\/remeds.org\/pl\/wp-json\/wp\/v2\/media?parent=3562"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/remeds.org\/pl\/wp-json\/wp\/v2\/categories?post=3562"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/remeds.org\/pl\/wp-json\/wp\/v2\/tags?post=3562"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}