{"id":15969,"date":"2022-06-14T20:42:45","date_gmt":"2022-06-14T18:42:45","guid":{"rendered":"https:\/\/dupmecp2.eu\/reversione-dei-fenotipi-in-modelli-di-topi-mecp2-usando-oligonucleotidi-antisenso\/"},"modified":"2023-04-17T15:46:43","modified_gmt":"2023-04-17T15:46:43","slug":"%ce%b1%ce%bd%ce%b1%cf%83%cf%84%cf%81%ce%bf%cf%86%ce%ae-%cf%84%cf%89%ce%bd-%cf%86%ce%b5%ce%bd%cf%8c%cf%84%ce%b9%cf%80%cf%89%ce%bd-%cf%83%ce%b5-%ce%bc%ce%bf%ce%bd%cf%84%ce%ad%ce%bb%ce%b1-%ce%ba%ce%bf","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/el\/%ce%b1%ce%bd%ce%b1%cf%83%cf%84%cf%81%ce%bf%cf%86%ce%ae-%cf%84%cf%89%ce%bd-%cf%86%ce%b5%ce%bd%cf%8c%cf%84%ce%b9%cf%80%cf%89%ce%bd-%cf%83%ce%b5-%ce%bc%ce%bf%ce%bd%cf%84%ce%ad%ce%bb%ce%b1-%ce%ba%ce%bf\/","title":{"rendered":"\"Reversione dei fenotipi in modelli di topi MECP2 usando oligonucleotidi antisenso\"."},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>\u0397 \u03ba\u03b1\u03b8\u03b7\u03b3\u03ae\u03c4\u03c1\u03b9\u03b1 Huda Zoghbi \u03ba\u03b1\u03b9 \u03bf\u03b9 \u03c3\u03c5\u03bd\u03b5\u03c1\u03b3\u03ac\u03c4\u03b5\u03c2 \u03c4\u03b7\u03c2 \u03c3\u03c4\u03bf Baylor College of Medicine \u03c3\u03c4\u03bf \u03a4\u03ad\u03be\u03b1\u03c2 \u03c4\u03c9\u03bd \u0397\u03a0\u0391 \u03b4\u03b7\u03bc\u03bf\u03c3\u03af\u03b5\u03c5\u03c3\u03b1\u03bd \u03ad\u03bd\u03b1 \u03ac\u03c1\u03b8\u03c1\u03bf \u03bc\u03b5 \u03c4\u03af\u03c4\u03bb\u03bf \"Reversibilita' del fenotipo nel topo utilizzando il salvataggio genetico o oligonucleotidi antisenso\". \u03a3\u03c4\u03bf \u03ac\u03c1\u03b8\u03c1\u03bf \u03b1\u03c5\u03c4\u03cc \u03c0\u03b1\u03c1\u03bf\u03c5\u03c3\u03b9\u03ac\u03b6\u03bf\u03bd\u03c4\u03b1\u03b9 \u03c4\u03b1 \u03b1\u03c0\u03bf\u03c4\u03b5\u03bb\u03ad\u03c3\u03bc\u03b1\u03c4\u03b1 \u03c4\u03b7\u03c2 \u03c7\u03c1\u03ae\u03c3\u03b7\u03c2 antisense \u03bf\u03bb\u03b9\u03b3\u03bf\u03bd\u03bf\u03c5\u03ba\u03bb\u03b5\u03bf\u03c4\u03b9\u03b4\u03af\u03c9\u03bd \u03b3\u03b9\u03b1 \u03c4\u03b7 \u03b4\u03b9\u03cc\u03c1\u03b8\u03c9\u03c3\u03b7 \u03c4\u03c9\u03bd \u03b5\u03c0\u03b9\u03c0\u03ad\u03b4\u03c9\u03bd \u03c4\u03bf\u03c5 MeCP2 \u03ba\u03b1\u03b9 \u03c4\u03b7\u03bd \u03c0\u03c1\u03cc\u03ba\u03bb\u03b7\u03c3\u03b7 \u03b1\u03bd\u03b1\u03c3\u03c4\u03c1\u03bf\u03c6\u03ae\u03c2 \u03c4\u03c9\u03bd \u03b4\u03b9\u03b1\u03c4\u03b1\u03c1\u03b1\u03c7\u03ce\u03bd \u03c3\u03c5\u03bc\u03c0\u03b5\u03c1\u03b9\u03c6\u03bf\u03c1\u03ac\u03c2, \u03bc\u03bf\u03c1\u03b9\u03b1\u03ba\u03ae\u03c2 \u03ba\u03b1\u03b9 \u03b7\u03bb\u03b5\u03ba\u03c4\u03c1\u03b9\u03ba\u03ae\u03c2 \u03bb\u03b5\u03b9\u03c4\u03bf\u03c5\u03c1\u03b3\u03af\u03b1\u03c2 \u03c3\u03b5 \u03b6\u03c9\u03b9\u03ba\u03ac \u03bc\u03bf\u03bd\u03c4\u03ad\u03bb\u03b1 \u03bc\u03b5 \u03c3\u03cd\u03bd\u03b4\u03c1\u03bf\u03bc\u03bf \u03b4\u03b9\u03c0\u03bb\u03b1\u03c3\u03b9\u03b1\u03c3\u03bc\u03bf\u03cd \u03c4\u03bf\u03c5 MECP2.<\/strong><\/p>\n\n<p class=\"wp-block-paragraph\">\u0391\u03bd\u03c4\u03b9\u03c3\u03c4\u03c1\u03bf\u03c6\u03ae \u03c4\u03c9\u03bd \u03c6\u03b1\u03b9\u03bd\u03bf\u03c4\u03cd\u03c0\u03c9\u03bd \u03c3\u03b5 \u03c0\u03bf\u03bd\u03c4\u03af\u03ba\u03b9\u03b1 \u03bc\u03b5 \u03b4\u03b9\u03c0\u03bb\u03b1\u03c3\u03b9\u03b1\u03c3\u03bc\u03cc \u03c4\u03bf\u03c5 MECP2 \u03bc\u03b5 \u03c4\u03b7 \u03c7\u03c1\u03ae\u03c3\u03b7 \u03b3\u03b5\u03bd\u03b5\u03c4\u03b9\u03ba\u03ae\u03c2 \u03b4\u03b9\u03ac\u03c3\u03c9\u03c3\u03b7\u03c2 \u03ae \u03b1\u03bd\u03c4\u03b9\u03c3\u03b7\u03bc\u03b9\u03ba\u03ce\u03bd \u03bf\u03bb\u03b9\u03b3\u03bf\u03bd\u03bf\u03c5\u03ba\u03bb\u03b5\u03bf\u03c4\u03b9\u03b4\u03af\u03c9\u03bd, Huda Zoghbi <em>et al,<\/em> <em>\u03a6\u03cd\u03c3\u03b7<\/em><strong> 528, <\/strong>123-126 (2015)<\/p>\n\n<p class=\"wp-block-paragraph\"><\/p>\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n<p class=\"wp-block-paragraph\"><\/p>\n\n<p class=\"wp-block-paragraph\"><strong>RIASSUNTO DELL'ARTICOLO:<\/strong><\/p>\n\n<p class=\"wp-block-paragraph\">\u039c\u03b9\u03b1 \u03c0\u03b1\u03c1\u03b1\u03bb\u03bb\u03b1\u03b3\u03ae \u03c3\u03c4\u03bf\u03bd \u03b1\u03c1\u03b9\u03b8\u03bc\u03cc \u03c4\u03c9\u03bd \u03b1\u03bd\u03c4\u03b9\u03b3\u03c1\u03ac\u03c6\u03c9\u03bd \u03c4\u03bf\u03c5 \u03b3\u03bf\u03bd\u03b9\u03b4\u03af\u03bf\u03c5 \u03ad\u03c7\u03b5\u03b9 \u03c3\u03c5\u03c7\u03bd\u03ac \u03c3\u03c5\u03c3\u03c7\u03b5\u03c4\u03b9\u03c3\u03c4\u03b5\u03af \u03bc\u03b5 \u03b1\u03bd\u03b1\u03c0\u03c4\u03c5\u03be\u03b9\u03b1\u03ba\u03ae \u03ba\u03b1\u03b8\u03c5\u03c3\u03c4\u03ad\u03c1\u03b7\u03c3\u03b7, \u03bd\u03bf\u03b7\u03c4\u03b9\u03ba\u03ae \u03b1\u03bd\u03b1\u03c0\u03b7\u03c1\u03af\u03b1 \u03ba\u03b1\u03b9 \u03b4\u03b9\u03b1\u03c4\u03b1\u03c1\u03b1\u03c7\u03ad\u03c2 \u03c0\u03bf\u03c5 \u03b1\u03bd\u03ae\u03ba\u03bf\u03c5\u03bd \u03c3\u03c4\u03bf \u03b1\u03c5\u03c4\u03b9\u03c3\u03c4\u03b9\u03ba\u03cc \u03c6\u03ac\u03c3\u03bc\u03b1. \u03a4\u03bf Sindrome Duplication MECP2 \u03b5\u03af\u03bd\u03b1\u03b9 \u03bc\u03af\u03b1 \u03b1\u03c0\u03cc \u03c4\u03b9\u03c2 \u03c0\u03b9\u03bf \u03c3\u03c5\u03c7\u03bd\u03ad\u03c2 \u03b3\u03bf\u03bd\u03b9\u03b4\u03b9\u03c9\u03bc\u03b1\u03c4\u03b9\u03ba\u03ad\u03c2 \u03b1\u03bd\u03b1\u03b4\u03b9\u03b1\u03c4\u03ac\u03be\u03b5\u03b9\u03c2 \u03c3\u03c4\u03b1 maschi \u03ba\u03b1\u03b9 \u03c7\u03b1\u03c1\u03b1\u03ba\u03c4\u03b7\u03c1\u03af\u03b6\u03b5\u03c4\u03b1\u03b9 \u03b1\u03c0\u03cc \u03b1\u03c5\u03c4\u03b9\u03c3\u03bc\u03cc, \u03b4\u03b9\u03b1\u03bd\u03bf\u03b7\u03c4\u03b9\u03ba\u03ae \u03b1\u03bd\u03b1\u03c0\u03b7\u03c1\u03af\u03b1, \u03ba\u03b9\u03bd\u03b7\u03c4\u03b9\u03ba\u03ad\u03c2 \u03b1\u03bd\u03b5\u03c0\u03ac\u03c1\u03ba\u03b5\u03b9\u03b5\u03c2, \u03ac\u03b3\u03c7\u03bf\u03c2, \u03b5\u03c0\u03b9\u03bb\u03b7\u03c8\u03af\u03b1, \u03c3\u03c5\u03c7\u03bd\u03ad\u03c2 \u03b1\u03bd\u03b1\u03c0\u03bd\u03b5\u03c5\u03c3\u03c4\u03b9\u03ba\u03ad\u03c2 \u03bb\u03bf\u03b9\u03bc\u03ce\u03be\u03b5\u03b9\u03c2 \u03ba\u03b1\u03b9 \u03c0\u03c1\u03cc\u03c9\u03c1\u03bf \u03b8\u03ac\u03bd\u03b1\u03c4\u03bf.<\/p>\n\n<p class=\"wp-block-paragraph\">\u03a4\u03bf \u03b5\u03c5\u03c1\u03cd \u03c6\u03ac\u03c3\u03bc\u03b1 \u03c4\u03c9\u03bd \u03b5\u03bb\u03bb\u03b5\u03b9\u03bc\u03bc\u03ac\u03c4\u03c9\u03bd \u03c0\u03bf\u03c5 \u03c0\u03c1\u03bf\u03ba\u03b1\u03bb\u03bf\u03cd\u03bd\u03c4\u03b1\u03b9 \u03b1\u03c0\u03cc \u03c4\u03b7\u03bd \u03c0\u03c1\u03c9\u03c4\u03b5\u0390\u03bd\u03b7 MECP2 \u03c0\u03bf\u03c5 \u03c0\u03b1\u03c1\u03ac\u03b3\u03b5\u03c4\u03b1\u03b9 \u03c3\u03b5 \u03c0\u03b5\u03c1\u03af\u03c3\u03c3\u03b5\u03b9\u03b1 \u03b1\u03c0\u03bf\u03c4\u03b5\u03bb\u03b5\u03af \u03bc\u03b5\u03b3\u03ac\u03bb\u03b7 \u03c0\u03c1\u03cc\u03ba\u03bb\u03b7\u03c3\u03b7 \u03b3\u03b9\u03b1 \u03c4\u03b9\u03c2 \u03c0\u03b1\u03c1\u03b1\u03b4\u03bf\u03c3\u03b9\u03b1\u03ba\u03ad\u03c2 \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03c5\u03c4\u03b9\u03ba\u03ad\u03c2 \u03c0\u03c1\u03bf\u03c3\u03b5\u03b3\u03b3\u03af\u03c3\u03b5\u03b9\u03c2 \u03c0\u03bf\u03c5 \u03b2\u03b1\u03c3\u03af\u03b6\u03bf\u03bd\u03c4\u03b1\u03b9 \u03c3\u03b5 \u03b2\u03b9\u03bf\u03c7\u03b7\u03bc\u03b9\u03ba\u03ad\u03c2 \u03b5\u03ba\u03c4\u03b9\u03bc\u03ae\u03c3\u03b5\u03b9\u03c2. Di conseguenza, abbiamo perseguito strategie che hanno come target direttamente la proteina MeCP2 e sono suscettibili di essere trasposte in una terapia clinica.<\/p>\n\n<p class=\"wp-block-paragraph\">La prima domanda a cui abbiamo cercato di dare una risposta e' se la disfunzione neurologica possa essere reversibile una volta che i sintomi si sono evidenziati. \u0397 \u03b1\u03bd\u03c4\u03b9\u03c3\u03c4\u03c1\u03bf\u03c6\u03ae \u03c4\u03bf\u03c5 \u03c6\u03b1\u03b9\u03bd\u03bf\u03bc\u03ad\u03bd\u03bf\u03c5 \u03c3\u03b5 \u03b5\u03bd\u03ae\u03bb\u03b9\u03ba\u03b5\u03c2 \u03b1\u03c3\u03b8\u03b5\u03bd\u03b5\u03af\u03c2 \u03bc\u03b5 \u03c3\u03b9\u03bd\u03ce\u03bc\u03bc\u03b1\u03c4\u03b1 \u03ad\u03c7\u03b5\u03b9 \u03b1\u03c0\u03bf\u03b4\u03b5\u03b9\u03c7\u03b8\u03b5\u03af \u03c3\u03b5 \u03bc\u03bf\u03bd\u03c4\u03ad\u03bb\u03b1 \u03bd\u03b5\u03c5\u03c1\u03bf\u03bb\u03bf\u03b3\u03b9\u03ba\u03ce\u03bd \u03b4\u03b9\u03b1\u03c4\u03b1\u03c1\u03b1\u03c7\u03ce\u03bd \u03c0\u03bf\u03c5 \u03bf\u03c6\u03b5\u03af\u03bb\u03bf\u03bd\u03c4\u03b1\u03b9 \u03c3\u03b5 \u03bc\u03bf\u03bd\u03bf\u03b3\u03bf\u03bd\u03b9\u03b4\u03b9\u03b1\u03ba\u03ae \u03b1\u03c0\u03ce\u03bb\u03b5\u03b9\u03b1 \u03bb\u03b5\u03b9\u03c4\u03bf\u03c5\u03c1\u03b3\u03af\u03b1\u03c2, \u03cc\u03c0\u03c9\u03c2 \u03b7 \u03b1\u03c0\u03ce\u03bb\u03b5\u03b9\u03b1 \u03c4\u03bf\u03c5 MeCP2 \u03c3\u03c4\u03bf Rett sindrome. Ci\u00f2 indica che, almeno in alcuni casi, la neuroanatomia pu\u00f2 rimanere sufficientemente intatta cosi' che la correzione della disfunzione molecolare alla base di questi disturbi possa ristabilire una fisiologia sana.<\/p>\n\n<p class=\"wp-block-paragraph\">Data l'assenza di neurodegenerazione nella Sindrome da Duplicazione di MECP2, noi proponiamo che il ripristino dei normali livelli di MeCP2 negli adulti di topi modello possa corregger il loro fenotipo.<\/p>\n\n<p class=\"wp-block-paragraph\">Creando e caratterizzando un modello di topo con proteina MeCP2 sovra espressa, dimostriamo che la correzione dei livelli di MeCP2 inverte in gran parte i deficit comportamentali, molecolari e elettrofisiologici.<\/p>\n\n<p class=\"wp-block-paragraph\">\u03a4\u03b1 \u03b5\u03c0\u03af\u03c0\u03b5\u03b4\u03b1 \u03c4\u03bf\u03c5 MeCP2 \u03ad\u03c7\u03bf\u03c5\u03bd \u03b4\u03b9\u03b1\u03b3\u03c1\u03b1\u03c6\u03b5\u03af \u03bc\u03b5 \u03c4\u03b7 \u03c7\u03c1\u03ae\u03c3\u03b7 \u03b5\u03bd\u03cc\u03c2 \u03b1\u03bd\u03c4\u03b9\u03b1\u03b9\u03c3\u03b8\u03b7\u03c4\u03b9\u03ba\u03bf\u03cd \u03bf\u03bb\u03b9\u03b3\u03bf\u03bd\u03bf\u03c5\u03ba\u03bb\u03b5\u03bf\u03c4\u03b9\u03b4\u03af\u03bf\u03c5.<\/p>\n\n<p class=\"wp-block-paragraph\">Queste molecole sono acidi nucleici modificati che possono ibridizzarsi selettivamente con l'RNA messaggero trascritto da un gene silenziandolo e sono gia' stati usati con successo in diversi modelli di topo.<\/p>\n\n<p class=\"wp-block-paragraph\">\u0394\u03b9\u03b1\u03c0\u03b9\u03c3\u03c4\u03ce\u03c3\u03b1\u03bc\u03b5 \u03cc\u03c4\u03b9 \u03b7 \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03af\u03b1 \u03bc\u03b5 \u03b1\u03bd\u03c4\u03b9\u03c3\u03b7\u03bc\u03b7\u03c4\u03b9\u03ba\u03ac \u03bf\u03bb\u03b9\u03b3\u03bf\u03bd\u03bf\u03c5\u03ba\u03bb\u03b5\u03bf\u03c4\u03af\u03b4\u03b9\u03b1 \u03c0\u03c1\u03bf\u03ba\u03b1\u03bb\u03b5\u03af \u03c3\u03b7\u03bc\u03b1\u03bd\u03c4\u03b9\u03ba\u03ae \u03b1\u03c0\u03bf\u03ba\u03b1\u03c4\u03ac\u03c3\u03c4\u03b1\u03c3\u03b7 \u03c4\u03bf\u03c5 \u03c6\u03b5\u03bd\u03cc\u03c4\u03b9\u03c0\u03bf\u03c5 \u03c3\u03b5 \u03b4\u03b9\u03b1\u03b3\u03bf\u03bd\u03b9\u03b4\u03b9\u03b1\u03ba\u03bf\u03cd\u03c2 \u03b5\u03bd\u03ae\u03bb\u03b9\u03ba\u03b5\u03c2 \u03bc\u03b5 \u03c3\u03c5\u03bc\u03c0\u03c4\u03ce\u03bc\u03b1\u03c4\u03b1 \u03c4\u03b7\u03c2 \u03bd\u03cc\u03c3\u03bf\u03c5 \u03c4\u03bf\u03c5 \u03b4\u03b9\u03c0\u03bb\u03b1\u03c3\u03b9\u03b1\u03c3\u03bc\u03bf\u03cd \u03c4\u03bf\u03c5 MECP2 (MECP2-TG) \u03ba\u03b1\u03b9 \u03b4\u03b9\u03bf\u03c1\u03b8\u03ce\u03bd\u03b5\u03b9 \u03c4\u03b1 \u03b5\u03c0\u03af\u03c0\u03b5\u03b4\u03b1 MeCP2 \u03c3\u03c4\u03b1 \u03bb\u03b9\u03bd\u03bf\u03b2\u03bb\u03b1\u03c3\u03c4\u03bf\u03b5\u03b9\u03b4\u03ae \u03ba\u03cd\u03c4\u03c4\u03b1\u03c1\u03b1 \u03b1\u03c3\u03b8\u03b5\u03bd\u03ce\u03bd \u03bc\u03b5 \u03bd\u03cc\u03c3\u03bf \u03c4\u03bf\u03c5 \u03b4\u03b9\u03c0\u03bb\u03b1\u03c3\u03b9\u03b1\u03c3\u03bc\u03bf\u03cd \u03c4\u03bf\u03c5 MECP2 \u03c3\u03b5 \u03c3\u03c7\u03ad\u03c3\u03b7 \u03bc\u03b5 \u03c4\u03b7 \u03b4\u03cc\u03c3\u03b7 \u03c4\u03c9\u03bd \u03c7\u03bf\u03c1\u03b7\u03b3\u03bf\u03cd\u03bc\u03b5\u03bd\u03c9\u03bd \u03bf\u03bb\u03b9\u03b3\u03bf\u03bd\u03bf\u03c5\u03ba\u03bb\u03b5\u03bf\u03c4\u03b9\u03b4\u03af\u03c9\u03bd.<\/p>\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n<p class=\"wp-block-paragraph\"><\/p>\n\n<div class=\"wp-block-buttons is-layout-flex wp-block-buttons-is-layout-flex\">\n<div class=\"wp-block-button is-style-fill\"><a class=\"wp-block-button__link has-white-color has-text-color has-background wp-element-button\" href=\"https:\/\/www.nature.com\/articles\/nature16159\" style=\"border-radius:14px;background-color:#f7a13f\" target=\"_blank\" rel=\"noreferrer noopener\">\u0394\u03b7\u03bc\u03bf\u03c3\u03af\u03b5\u03c5\u03c3\u03b7<\/a><\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"<p>La Prof.ssa Huda Zoghbi e collaboratori al Baylor College of Medicine in Texas, USA hanno pubblicato un articolo intitolato \u201cReversibilita\u2019 del fenotipo nel topo utilizzando il salvataggio genetico o oligonucleotidi [&hellip;]<\/p>","protected":false},"author":1,"featured_media":13095,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[139],"tags":[],"class_list":["post-15969","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-pubblicazioni"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v25.8 - 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