{"id":12020,"date":"2021-01-06T15:09:42","date_gmt":"2021-01-06T20:09:42","guid":{"rendered":"https:\/\/www.progeriaresearch.org\/?p=12020"},"modified":"2022-11-14T17:14:57","modified_gmt":"2022-11-14T22:14:57","slug":"kicking-off-the-new-year-with-exciting-research-news","status":"publish","type":"post","link":"https:\/\/www.progeriaresearch.org\/it\/2021\/01\/06\/kicking-off-the-new-year-with-exciting-research-news\/","title":{"rendered":"Iniziamo il nuovo anno con entusiasmanti novit\u00e0 sulla ricerca!"},"content":{"rendered":"

[et_pb_section fb_built=”1″ _builder_version=”4.16″ custom_padding=”0px|||||” global_colors_info=”{}”][et_pb_row _builder_version=”4.16″ background_size=”initial” background_position=”top_left” background_repeat=”repeat” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”content base” _builder_version=”4.16″ background_size=”initial” background_position=”top_left” background_repeat=”repeat” global_colors_info=”{}”]<\/p>\n

\"\"<\/p>\n

Felice anno nuovo! Ci auguriamo che tutti abbiano trascorso delle vacanze sane e riposanti.<\/p>\n

Iniziamo il 2021 con altre interessanti novit\u00e0 sulla ricerca<\/strong>A gennaio, la rivista scientifica Natura<\/em> pubblicati risultati rivoluzionari che dimostrano che l'editing genetico in un modello murino di Progeria ha corretto la mutazione che causa la Progeria in molte cellule,<\/strong> ha migliorato diversi sintomi chiave della malattia e durata della vita notevolmente aumentata<\/strong> nei topi.<\/p>\n

Cofinanziato da PRF e co-redatto dal Direttore medico di PRF, il dott. Leslie Gordon, lo studio ha scoperto che con una singola iniezione di un editor di base programmato per correggere la mutazione che causa la malattia, i topi sopravvivevano 2,5 volte pi\u00f9 a lungo dei topi di controllo con Progeria non trattati, fino a un'et\u00e0 corrispondente all'inizio della vecchiaia nei topi sani. \u00c8 importante notare che i topi trattati hanno anche mantenuto tessuto vascolare sano, una scoperta significativa, poich\u00e9 la perdita di integrit\u00e0 vascolare \u00e8 un predittore di mortalit\u00e0 nei bambini con Progeria.<\/p>\n

Lo studio \u00e8 stato co-diretto da un esperto mondiale di editing genetico, Dott. David Liu<\/a>del Broad Institute, MIT, Cambridge, MA, Jonathan Brown, professore associato di medicina presso la Divisione di medicina cardiovascolare presso la Vanderbilt University, e Francis Collins, MD, PhD, direttore dei National Institutes of Health.<\/p>\n

[\/et_pb_text][et_pb_testimonial author=”Dr. Francis Collins” job_title=”Director of the National Institutes of Health. ” admin_label=”Dr. Francis Collins” _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”]To see this dramatic response in our Progeria mouse model is one of the most exciting therapeutic developments I have been part of in 40 years as a physician-scientist,\u201d said Dr. Collins.
\n[\/et_pb_testimonial][et_pb_testimonial author=”David Lui, PhD” job_title=”Broad Institute, MIT” admin_label=”David Lui, PhD” _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”]Five years ago, we were still finishing the development of the very first base editor,\u201d said Dr. Liu. \u201cIf you had told me then that within five years, a single dose of a base editor could address Progeria in an animal at the DNA, RNA, protein, vascular pathology, and lifespan levels, I would have said \u2018there\u2019s no way.\u2019 It’s a real testament to the dedication of the team that made this work possible.
\n[\/et_pb_testimonial][et_pb_text _builder_version=”4.18.1″ _module_preset=”default” hover_enabled=”0″ global_colors_info=”{}” sticky_enabled=”0″]<\/p>\n

Sono necessari ulteriori studi preclinici per analizzare questi risultati, che speriamo un giorno porteranno a una sperimentazione clinica. Non vediamo l'ora di tenervi informati su questa entusiasmante scoperta man mano che si faranno ulteriori progressi!<\/p>\n

Leggi di pi\u00f9 su questa entusiasmante notizia Qui<\/a>.<\/p>\n

[\/et_pb_text][et_pb_text _builder_version=”4.18.1″ _module_preset=”default” hover_enabled=”0″ global_colors_info=”{}” sticky_enabled=”0″]<\/p>\n

\n

Mentre entriamo nel 2021, siamo grati a partner come TE, che rendono possibile questo tipo di progresso.<\/p>\n

L'anno scorso solo<\/em>, La Fondazione per la ricerca sulla Progeria:<\/strong><\/span><\/p>\n

\n

    \n
  • \u00c8 stato raggiunto un traguardo importante con il primo trattamento per la Progeria approvato dalla FDA, il lonafarnib;<\/li>\n
  • Ha portato bambini affetti da Progeria da tutto il mondo al Boston Children's Hospital per la sperimentazione clinica finanziata dalla PRF (quando i viaggi erano consentiti a causa del COVID-19);<\/li>\n
  • Tenuto il nostro 10\u00b0 Workshop scientifico internazionale sulla progeria<\/em>, riunendo un numero record di 370 partecipanti da 30 paesi nel nostro primo incontro scientifico basato sul web;<\/li>\n
  • Continuiamo con impegno con le nostre principali attivit\u00e0 programmatiche che ci condurranno ai trattamenti e alla cura del futuro!<\/li>\n<\/ul>\n

    [\/et_pb_text][et_pb_text _builder_version=”4.18.1″ _module_preset=”default” hover_enabled=”0″ global_colors_info=”{}” sticky_enabled=”0″]<\/p>\n

    \n

    Koblan LW, Erdos MR, Wilson C, Cabral\u00a0<\/sup>WA, Levy JM, Iong, ZM, Tavarez\u00a0<\/sup>UL, Davison L, Gete YG, Mao X, Newby\u00a0<\/sup>GA, Doherty SP, Narisu N, Sheng Q, Krilow C, Lin CY,\u00a0Gordon<\/b>\u00a0LB, Cao K, Collins FS, Marrone JD, Liu\u00a0DOTTORE\u00a0<\/span>In vivo\u00a0<\/i>L'editing delle basi dell'adenina salva la sindrome di Hutchinson-Gilford dalla progeria\u00a0<\/b>Natura. 6 gennaio 2021<\/span><\/p>\n

    [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section]<\/p>","protected":false},"excerpt":{"rendered":"

    A gennaio, la rivista scientifica Natura<\/i> hanno pubblicato risultati rivoluzionari che dimostrano che l'editing genetico in un modello murino di Progeria ha corretto la mutazione che causa la Progeria in molte cellule, ha migliorato diversi sintomi chiave della malattia e ha aumentato notevolmente la durata della vita nei topi.<\/p>","protected":false},"author":2,"featured_media":12024,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_et_pb_use_builder":"on","_et_pb_old_content":"","_et_gb_content_width":"","footnotes":"","_links_to":"","_links_to_target":""},"categories":[2,1],"tags":[],"class_list":["post-12020","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news","category-uncategorized"],"yoast_head":"\nKicking off the New Year with exciting research news! - The Progeria Research Foundation<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, 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