{"id":3505,"date":"2025-06-13T20:45:29","date_gmt":"2025-06-13T20:45:29","guid":{"rendered":"https:\/\/life4hsp.com\/?p=3505"},"modified":"2025-06-23T11:05:38","modified_gmt":"2025-06-23T09:05:38","slug":"approvata-la-prima-sperimentazione-di-una-nuova-terapia-genica-per-la-hsp-spg47","status":"publish","type":"post","link":"https:\/\/life4hsp.com\/it\/approvata-la-prima-sperimentazione-di-una-nuova-terapia-genica-per-la-hsp-spg47\/","title":{"rendered":"Approvata la prima sperimentazione di una nuova terapia genica per la HSP (SPG47)"},"content":{"rendered":"<p><span data-olk-copy-source=\"MessageBody\">Ci sono notizie incoraggianti per le persone affette da HSP: l'autorit\u00e0 sanitaria statunitense FDA ha approvato una prima sperimentazione di una nuova terapia genica per una rara forma di paraplegia spastica ereditaria, la SPG47.<\/span><\/p>\n<h3><b>Che cos'\u00e8 l'SPG47?<\/b><\/h3>\n<p>L\u2019SPG47 \u00e8 una forma rara ed ereditaria di HSP che si manifesta gi\u00e0 nell\u2019infanzia con sintomi come rigidit\u00e0 muscolare, difficolt\u00e0 a camminare, ritardi nello sviluppo e problemi di linguaggio. La causa \u00e8 una mutazione del gene SPG47, che impedisce il corretto funzionamento di una proteina fondamentale nelle cellule cerebrali. Attualmente non esiste alcun trattamento disponibile.<\/p>\n<h3><b>Cosa succeder\u00e0 ora?<\/b><\/h3>\n<p>Un\u2019azienda biotecnologica <a href=\"https:\/\/blackfin.bio\/\">BlackfinBio<\/a>ha ricevuto il via libera per avviare un primo studio su bambini affetti da SPG47. Lo studio si svolger\u00e0 in un ospedale specializzato a Boston (USA). L\u2019obiettivo \u00e8 verificare se la terapia \u00e8 sicura.<\/p>\n<p>Il trattamento consiste in un\u2019unica iniezione nel liquido cerebrospinale contenente un frammento di DNA \u201csano\u201d. Questo gene corretto dovrebbe permettere alle cellule del bambino di produrre di nuovo la proteina mancante.<\/p>\n<h3><b>Perch\u00e9 \u00e8 importante?<\/b><\/h3>\n<ul>\n<li>\u00c8 il primo trattamento per SPG47 testato sull\u2019uomo.<\/li>\n<li>La terapia agisce sulla causa della malattia, non solo sui sintomi.<\/li>\n<li>L\u2019approvazione della FDA rappresenta un grande passo verso future terapie.<\/li>\n<\/ul>\n<h3><b>Cosa significa per le famiglie?<\/b><\/h3>\n<p>Anche se lo studio \u00e8 ancora nelle fasi iniziali, rappresenta una nuova speranza per i bambini con SPG47 e le loro famiglie. Si stanno facendo progressi e scienziati e medici stanno lavorando seriamente a soluzioni per le forme rare di HSP. <a href=\"https:\/\/www.sheffield.ac.uk\/news\/fda-approves-trial-advance-pioneering-treatment-hereditary-spastic-paraplegia-hsp\">clicca qui per<\/a> sulla ricerca<\/p>","protected":false},"excerpt":{"rendered":"<p>Er is hoopvol nieuws voor mensen met HSP: de Amerikaanse gezondheidsautoriteit FDA heeft toestemming gegeven voor een eerste proef met een nieuwe gentherapie voor een zeldzame vorm van erfelijke spastische paraplegie, namelijk SPG47. Wat is SPG47? SPG47 is een zeldzame, erfelijke vorm van HSP waarbij kinderen al op jonge leeftijd klachten krijgen zoals spierstijfheid, moeite [&hellip;]<\/p>\n","protected":false},"author":5,"featured_media":3506,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"inline_featured_image":false,"footnotes":""},"categories":[13],"tags":[],"class_list":["post-3505","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-onderzoek"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.9 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Gentherapie voor SPG47<\/title>\n<meta name=\"description\" content=\"de Amerikaanse gezondheidsautoriteit FDA heeft toestemming gegeven voor een eerste proef met een nieuwe gentherapie voor SPG47.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" 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