{"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":"reversibler-phanotyp-in-humanisiertem-mausmodell","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/de\/reversibler-phanotyp-in-humanisiertem-mausmodell\/","title":{"rendered":"Reversibler Ph\u00e4notyp in humanisiertem Mausmodell"},"content":{"rendered":"<p><strong>Shao et al. from Prof. Zoghbi's group (Baylor College of Medicine, Texas, USA) published an article in Science Translational medicine entitled \"Antisense oligonucleotide therapy in a humanized mouse model of MECP2 duplication syndrome\" (Antisense-Oligonucleotid-Therapie in einem humanisierten Mausmodell des MECP2-Duplikationssyndroms). <\/strong><\/p>\n\n\n\n<p><strong>Jede Spezies hat ihre eigenen Gene. Die Gene codieren f\u00fcr Proteine, die f\u00fcr jede Art einzigartig sind. Es ist m\u00f6glich, dass sich die Proteine von Mensch und Maus leicht voneinander unterscheiden. The model used in the presented work, called \"humanized model\", allows to evaluate more the activity of antisense oligonucleotides (ASO) specifically on the human protein produced by a mouse. Es ist daher n\u00e4her an der zuk\u00fcnftigen Verwendung von ASOs beim Menschen.<\/strong><\/p>\n\n\n\n<p><strong>Dieser Artikel stellt die Ergebnisse vor, die nach intracerebroventricularer Injektion von Antisense-Oligonucleotiden in diesem Mausmodell der \"humanisierten MECP2\"-Genduplikation erzielt wurden, in dem die Maus zwei menschliche MECP2-Allele und keine endogenen Mausallele tr\u00e4gt.<\/strong><\/p>\n\n\n\n<p><strong>Administration reduced effectively MeCP2 expression throughout the brains of these mice, alleviated several behavioral deficits, and restored the expression of some MeCP2-regulated genes in a dose-dependent manner and without any toxicity.<\/strong><\/p>\n\n\n\n<p>Antisense oligonucleotide therapy in a humanized mouse model of MECP2 duplication syndrome, Shao et al.<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>VER\u00d6FFENTLICHUNG ABSTRACT<\/strong><\/p>\n\n\n\n<p>Viele geistige Behinderungen sind auf Kopienzahlvariationen zur\u00fcckzuf\u00fchren, und bislang wurden keine Behandlungsm\u00f6glichkeiten f\u00fcr diese Klasse von Krankheiten getestet. Das MECP2-Duplikationssyndrom (MDS) ist eine der h\u00e4ufigsten genomischen Umlagerungen bei M\u00e4nnern und resultiert aus Duplikationen, die den Methyl-CpG-Bindungsprotein 2 (MECP2)-Genort umfassen. Wir haben zuvor gezeigt, dass die Therapie mit Antisense-Oligonukleotiden (ASO) die Menge des MeCP2-Proteins in einem MDS-Mausmodell reduzieren und seine Krankheitsmerkmale umkehren kann.<\/p>\n\n\n\n<p>Dieses MDS-Mausmodell trug jedoch ein transgenes humanes Allel und ein Maus-Allel, wobei letzteres vor dem human-spezifischen MECP2-ASO-Targeting gesch\u00fctzt war.<\/p>\n\n\n\n<p>Da MeCP2 ein dosissensitives Protein ist, muss die ASO so titriert werden, dass die Menge an MeCP2 nicht zu weit reduziert wird, was das Rett-Syndrom verursachen w\u00fcrde. Daher erzeugten wir ein \"MECP2 humanisiertes\" MDS-Modell, das zwei menschliche MECP2-Allele und kein museales endogenes Allel tr\u00e4gt. Intracerebroventricular injection of the MECP2-ASO efficiently down-regulated MeCP2 expression throughout the brain in these mice. Moreover, MECP2-ASO mitigated several behavioral deficits and restored expression of selected MeCP2-regulated genes in a dose-dependent manner without any toxicity. Die Verabreichung von MECP2-ASO an das zentrale Nervensystem ist daher in diesem Mausmodell gut vertr\u00e4glich und vorteilhaft und stellt einen \u00fcbertragbaren Ansatz dar, der f\u00fcr die Behandlung von MDS praktikabel sein k\u00f6nnte.<\/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\">Zur Ver\u00f6ffentlichung<\/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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