{"id":2113,"date":"2006-02-16T20:28:04","date_gmt":"2006-02-16T20:28:04","guid":{"rendered":"https:\/\/www.progeriaresearch.org\/?p=2113"},"modified":"2020-12-09T11:16:33","modified_gmt":"2020-12-09T16:16:33","slug":"three-studies-released-that-bring-us-closer-than-ever-to-understanding-progeria-and-to-disease-treatment","status":"publish","type":"post","link":"https:\/\/www.progeriaresearch.org\/it\/2006\/02\/16\/three-studies-released-that-bring-us-closer-than-ever-to-understanding-progeria-and-to-disease-treatment\/","title":{"rendered":"Pubblicati tre studi che ci avvicinano pi\u00f9 che mai alla comprensione della Progeria e al trattamento della malattia"},"content":{"rendered":"

Italiano: [et_pb_section fb_built=\u201d1\u2033 _builder_version=\u201d3.22\u2033][et_pb_row _builder_version=\u201d3.25\u2033 background_size=\u201diniziale\u201d background_position=\u201dtop_left\u201d background_repeat=\u201dripeti\u201d][et_pb_column type=\u201d4_4\u2033 _builder_version=\u201d3.25\u2033 custom_padding=\u201d|||\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text _builder_version=\u201d4.6.5\u2033 background_size=\u201diniziale\u201d background_position=\u201dtop_left\u201d background_repeat=\u201dripeti\u201d hover_enabled=\u201d0\u2033 sticky_enabled=\u201d0\u2033]<\/p>\n

I ricercatori della UCLA, finanziati dal PRF, hanno preso un modello di topo simile alla Progeria e hanno testato un potenziale trattamento farmacologico per i bambini con Progeria. Il loro studio, pubblicato su Science il 16 febbraio, ha scoperto che questo farmaco FTI migliora alcuni segni della malattia.<\/p>\n

A settembre la Progeria Research Foundation \u00e8 stata lieta di annunciare che i ricercatori finanziati dalla PRF avevano pubblicato studi a sostegno di un potenziale trattamento farmacologico per i bambini affetti da Progeria: le cellule della Progeria si sono normalizzate (in una capsula di Petri) quando \u00e8 stato somministrato questo farmaco. La fase successiva dei test \u00e8 stata sui modelli animali e siamo entusiasti di annunciare che uno studio pubblicato su Science il 16 febbraio ha scoperto che questo farmaco FTI migliora alcuni segni della malattia in un modello murino simile alla progeria. La PRF ha contribuito a finanziare i ricercatori della UCLA, il dott. Loren Fong e il dott. Stephen Young, per questo ultimo, fondamentale passo verso le sperimentazioni cliniche sui bambini. Clicca qui per maggiori dettagli<\/a> su questa entusiasmante notizia.<\/p>\n

E le pubblicazioni scientifiche continuano! PRF ha finanziato uno studio della Dott. ssa Karima Djabali, insieme al Dott. Dayle McClintock e al Direttore medico PRF Dott. Leslie B. Gordon, che \u00e8 stato appena pubblicato nel PNAS di questa settimana (Atti dell'Accademia nazionale delle scienze).<\/em> Lo studio conclude che la proteina difettosa prodotta dal gene Progeria (chiamata progerina) si accumula nelle cellule delle pareti vascolari dei bambini. Questo ci mostra, per la prima volta, che esiste una connessione diretta tra progerina e malattie cardiache.<\/p>\n

E ultimo ma non meno importante, il laboratorio del Dott. Francis Collins, che ha scoperto il gene della Progeria, ha fatto un passo avanti e ha prodotto un topo Progeria che porta un difetto genetico identico a quello dei bambini. Questo classico modello di topo Progeria mostra una grave malattia vascolare e sar\u00e0 fondamentale per comprendere non solo come i bambini con Progeria sviluppano malattie cardiache, ma ancora pi\u00f9 importante sar\u00e0 un modello eccellente per testare nuovi trattamenti come FTI e terapie genetiche nel trattamento e nella cura della Progeria. E questo modello pu\u00f2 anche essere utilizzato per esplorare le malattie cardiovascolari in generale. Siamo orgogliosi di dire che il nostro Direttore medico, il Dott. Leslie B. Gordon, \u00e8 un coautore. Lo studio appare nel PNAS di questa settimana.<\/p>\n

Copie di entrambi gli articoli PNAS possono essere trovate sul sito web dei Proceedings of the National Academies of Science all'indirizzo www.pnas.org<\/a><\/p>\n

Tre studi in un mese: WOW! Stiamo davvero lavorando per la cura. Grazie a tutti quei ricercatori che lavorano duramente ogni giorno per aiutare i bambini con la progeria e i milioni di persone che soffrono di malattie cardiache in tutto il mondo. E grazie alle famiglie e ai bambini, e a tutti i nostri donatori per aver reso tutto questo possibile.<\/strong><\/p>\n

INSIEME, NOI VOLERE<\/em> TROVA LA CURA!<\/strong><\/p>\n

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[et_pb_section fb_built=”1″ _builder_version=”3.22″][et_pb_row _builder_version=”3.25″ background_size=”initial” background_position=”top_left” background_repeat=”repeat”][et_pb_column type=”4_4″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_text _builder_version=”4.6.5″ background_size=”initial” background_position=”top_left” background_repeat=”repeat” hover_enabled=”0″ sticky_enabled=”0″] PRF-funded, UCLA researchers have taken a Progeria-like mouse model and tested a potential drug treatment for children with Progeria. Their study released in Science Feb.16th has found that this FTI drug improves some signs of the disease. In […]<\/p>","protected":false},"author":1,"featured_media":2043,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_et_pb_use_builder":"on","_et_pb_old_content":"

[vc_custom_heading text=\"Three studies released that bring us closer than ever to understanding Progeria and to disease treatment\" font_container=\"tag:h1|text_align:left\" use_theme_fonts=\"yes\"]PRF-funded, UCLA researchers have taken a Progeria-like mouse model and tested a potential drug treatment for children with Progeria. Their study released in Science Feb.16th has found that this FTI drug improves some signs of the disease.<\/p>

In September The Progeria Research Foundation was pleased to announce that PRF-funded researchers had published studies supporting a potential drug treatment for children with Progeria - Progeria cells became normalized (in a dish) when given this drug. The next phase of testing has been in animal models, and we are excited to report that a study released in Science Feb.16th has found that this FTI drug improves some signs of disease in a progeria-like mouse model. PRF helped fund UCLA researchers Dr. Loren Fong and Dr. Stephen Young for this latest, vital step towards clinical treatment trials with the children. Click here for more details<\/a> on this exciting news.<\/p>

And the scientific publications continue! PRF funded a study by Dr. Karima Djabali, along with Dr. Dayle McClintock and PRF Medical Director Dr. Leslie B. Gordon, which was just published in this week's PNAS (Proceeding of the National Academy of Sciences).<\/em> The study concludes that the defective protein produced by the Progeria gene (called progerin) builds up in cells of the children's vessel walls. This shows us, for the first tine, that there is a direct connection between progerin and heart disease.<\/p>

And last but certainly not least, the laboratory of Dr. Francis Collins, who discovered the gene for Progeria, has forged ahead and produced a Progeria mouse that carries a genetic defect identical to that of the children. This classic Progeria mouse model shows severe vascular disease, and will be pivotal to our understanding not only how children with Progeria develop heart disease, but even more importantly will be excellent models for testing new treatments such as FTI and genetic therapies in treating and curing Progeria, And this model can also be used to explore cardiovascular disease in general. We are proud to say that our Medical Director, Dr. Leslie B. Gordon, is a co-author. The study appears in this week's PNAS.<\/p>

Copies of both PNAS articles can be found at the website of the Proceedings of the National Academies of Science at www.pnas.org<\/a><\/p>

Three studies in one month - WOW! We are really cooking towards the cure. Thank you to all of those researchers who are working hard every day to help children with Progeria and the millions of people who suffer from heart disease throughout the world. And thanks to the families and children, and all of our donors for making this all possible.<\/strong><\/p>

TOGETHER, WE WILL<\/em> FIND THE CURE!<\/strong><\/p>","_et_gb_content_width":"","footnotes":"","_links_to":"","_links_to_target":""},"categories":[2],"tags":[],"class_list":["post-2113","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news"],"yoast_head":"\nThree studies released that bring us closer than ever to understanding Progeria and to disease treatment - The Progeria Research Foundation<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.progeriaresearch.org\/it\/2006\/02\/16\/three-studies-released-that-bring-us-closer-than-ever-to-understanding-progeria-and-to-disease-treatment\/\" \/>\n<meta property=\"og:locale\" content=\"it_IT\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Three studies released that bring us closer than ever to understanding Progeria and to disease treatment - The Progeria Research Foundation\" \/>\n<meta property=\"og:description\" content=\"[et_pb_section fb_built=”1″ _builder_version=”3.22″][et_pb_row _builder_version=”3.25″ background_size=”initial” background_position=”top_left” background_repeat=”repeat”][et_pb_column type=”4_4″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_text _builder_version=”4.6.5″ background_size=”initial” background_position=”top_left” background_repeat=”repeat” hover_enabled=”0″ sticky_enabled=”0″] PRF-funded, UCLA researchers have taken a Progeria-like mouse model and tested a potential drug treatment for children with Progeria. 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