{"id":9344,"date":"2022-06-14T20:42:45","date_gmt":"2022-06-14T20:42:45","guid":{"rendered":"https:\/\/dupmecp2.eu\/reversible-phenotype-in-humanized-mouse-model\/"},"modified":"2023-01-13T11:44:59","modified_gmt":"2023-01-13T11:44:59","slug":"odwracalny-fenotyp-w-humanizowanym-modelu-mysim","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/pl\/odwracalny-fenotyp-w-humanizowanym-modelu-mysim\/","title":{"rendered":"Odwracalny fenotyp w humanizowanym modelu mysim"},"content":{"rendered":"<p><strong>Shao i wsp. z grupy prof. Zoghbi (Baylor College of Medicine, Teksas, USA) opublikowali artyku\u0142 w Science Translational medicine zatytu\u0142owany \"Antisense oligonucleotide therapy in a humanized mouse model of MECP2 duplication syndrome\". <\/strong><\/p>\n\n\n\n<p><strong>Ka\u017cdy gatunek ma swoje w\u0142asne geny. Geny koduj\u0105 bia\u0142ka, kt\u00f3re s\u0105 unikalne dla ka\u017cdego gatunku. Mo\u017cliwe jest, \u017ce bia\u0142ka ludzkie i mysie r\u00f3\u017cni\u0105 si\u0119 nieznacznie. Model zastosowany w prezentowanej pracy, zwany \"modelem humanizowanym\", pozwala lepiej oceni\u0107 aktywno\u015b\u0107 oligonukleotyd\u00f3w antysensownych (ASO) specyficznie na ludzkim bia\u0142ku wytwarzanym przez mysz. Jest zatem bli\u017cszy przysz\u0142emu zastosowaniu ASO u ludzi.<\/strong><\/p>\n\n\n\n<p><strong>W artykule przedstawiono wyniki uzyskane po dokomorowym wstrzykni\u0119ciu oligonukleotyd\u00f3w antysensownych w mysim modelu \"humanizowanej duplikacji genu MECP2\", w kt\u00f3rym mysz jest nosicielem dw\u00f3ch ludzkich alleli MECP2 i nie ma endogennych alleli myszy.<\/strong><\/p>\n\n\n\n<p><strong>Podanie skutecznie zmniejszy\u0142o ekspresj\u0119 MeCP2 w m\u00f3zgach tych myszy, z\u0142agodzi\u0142o kilka deficyt\u00f3w behawioralnych i przywr\u00f3ci\u0142o ekspresj\u0119 niekt\u00f3rych gen\u00f3w regulowanych przez MeCP2 w spos\u00f3b zale\u017cny od dawki i bez \u017cadnej toksyczno\u015bci.<\/strong><\/p>\n\n\n\n<p>Terapia oligonukleotydami antysensownymi w humanizowanym mysim modelu zespo\u0142u duplikacji MECP2, Shao i in.<em>Sci. Transl. Med<\/em>. <strong>13<\/strong>, 7785 (2021)<\/p>\n\n\n\n<p><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p><\/p>\n\n\n\n<p><strong>STRESZCZENIE PUBLIKACJI<\/strong><\/p>\n\n\n\n<p>Wiele zaburze\u0144 zwi\u0105zanych z niepe\u0142nosprawno\u015bci\u0105 intelektualn\u0105 jest spowodowanych zmianami liczby kopii i do tej pory nie przetestowano \u017cadnych opcji leczenia tej klasy chor\u00f3b. Zesp\u00f3\u0142 duplikacji MECP2 (MDS) jest jedn\u0105 z najcz\u0119stszych rearan\u017cacji genomowych u m\u0119\u017cczyzn i wynika z duplikacji obejmuj\u0105cych locus genu bia\u0142ka wi\u0105\u017c\u0105cego metylo-CpG 2 (MECP2). Wcze\u015bniej wykazali\u015bmy, \u017ce terapia oligonukleotydami antysensownymi (ASO) mo\u017ce zmniejszy\u0107 ilo\u015b\u0107 bia\u0142ka MeCP2 w mysim modelu MDS i odwr\u00f3ci\u0107 jego cechy chorobowe.<\/p>\n\n\n\n<p>Ten mysi model MDS zawiera\u0142 jednak jeden transgeniczny allel ludzki i jeden allel mysi, przy czym ten drugi by\u0142 chroniony przed specyficznym dla cz\u0142owieka celowaniem MECP2-ASO.<\/p>\n\n\n\n<p>Poniewa\u017c MeCP2 jest bia\u0142kiem wra\u017cliwym na dawk\u0119, ASO musi by\u0107 miareczkowane tak, aby ilo\u015b\u0107 MeCP2 nie zosta\u0142a zbytnio zmniejszona, co spowodowa\u0142oby zesp\u00f3\u0142 Retta. Dlatego wygenerowali\u015bmy \"humanizowany\" model MDS MECP2, kt\u00f3ry jest nosicielem dw\u00f3ch ludzkich alleli MECP2 i nie ma endogennego allelu myszy. Wewn\u0105trzm\u00f3zgowe wstrzykni\u0119cie MECP2-ASO skutecznie obni\u017cy\u0142o ekspresj\u0119 MeCP2 w m\u00f3zgu tych myszy. Co wi\u0119cej, MECP2-ASO z\u0142agodzi\u0142 kilka deficyt\u00f3w behawioralnych i przywr\u00f3ci\u0142 ekspresj\u0119 wybranych gen\u00f3w regulowanych przez MeCP2 w spos\u00f3b zale\u017cny od dawki bez \u017cadnej toksyczno\u015bci. Podawanie MECP2-ASO do o\u015brodkowego uk\u0142adu nerwowego jest zatem dobrze tolerowane i korzystne w tym modelu mysim i zapewnia mo\u017cliwe do prze\u0142o\u017cenia podej\u015bcie, kt\u00f3re mo\u017ce by\u0107 wykonalne w leczeniu MDS.<\/p>\n\n\n\n<div style=\"height:100px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\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-white-color has-text-color has-background wp-element-button\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/33658357\/\" style=\"border-radius:14px;background-color:#f7a13f\" target=\"_blank\" rel=\"noreferrer noopener\">Do publikacji<\/a><\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"<p>Shao et al. from Prof. Zoghbi&rsquo;s group (Baylor College of Medicine, Texas, USA) published an article in Science Translational medicine entitled \u00ab\u00a0Antisense oligonucleotide therapy in a humanized mouse model of [&hellip;]<\/p>","protected":false},"author":1,"featured_media":11723,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[88],"tags":[],"class_list":["post-9344","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-publications-en"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v25.8 - 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